{"id":"cholic-acid","rwe":[{"pmid":"41895615","year":"2026","title":"Altered serum 3β-hydroxy bile acids in major depressive disorder and associations with symptom dimensions.","finding":"","journal":"Journal of affective disorders","studyType":"Clinical Study"},{"pmid":"41887383","year":"2026","title":"The mechanism of Tibetan medicine Shibawei Niuhuang Qinggan pills in protecting against acetaminophen-induced acute liver injury in mice by regulating the Keap1-Nrf2 pathway.","finding":"","journal":"Journal of ethnopharmacology","studyType":"Clinical Study"},{"pmid":"41880860","year":"2026","title":"Usefulness of plasma bile acid profile as a prognostic biomarker for drug-induced liver injury.","finding":"","journal":"EBioMedicine","studyType":"Clinical Study"},{"pmid":"41869696","year":"2026","title":"Bilberry (Vaccinium myrtillus L.) extract alleviates gestational diabetes mellitus by coordinating TGR5/FXR signaling via a gut microbiota-bile acid axis.","finding":"","journal":"Food & function","studyType":"Clinical Study"},{"pmid":"41859732","year":"2026","title":"Autosomal recessive ELOVL1-related disorder presenting with severe neonatal cholestasis: A novel clinical feature?","finding":"","journal":"Molecular genetics and metabolism reports","studyType":"Clinical Study"}],"_fda":{"id":"edddbf7e-e2dd-4ff7-80a0-d73c22fd40ce","set_id":"5c6e4c9d-b85e-493f-9420-ab2fd0ff3723","openfda":{"nui":["N0000175802","M0002475"],"unii":["G1JO7801AE"],"route":["ORAL"],"rxcui":["1440861","1440868","1608274","1608276"],"spl_id":["edddbf7e-e2dd-4ff7-80a0-d73c22fd40ce"],"brand_name":["CHOLBAM"],"spl_set_id":["5c6e4c9d-b85e-493f-9420-ab2fd0ff3723"],"package_ndc":["79378-001-02","79378-002-02"],"product_ndc":["79378-001","79378-002"],"generic_name":["CHOLIC ACID"],"product_type":["HUMAN PRESCRIPTION DRUG"],"pharm_class_cs":["Bile Acids and Salts [CS]"],"substance_name":["CHOLIC ACID"],"pharm_class_epc":["Bile Acid [EPC]"],"manufacturer_name":["Mirum Pharmaceuticals Inc."],"application_number":["NDA205750"],"is_original_packager":[true]},"version":"2","pregnancy":["8.1 Pregnancy Pregnancy Surveillance Program There is a pregnancy surveillance program that monitors pregnancy outcomes in women exposed to CHOLBAM during pregnancy. Women who become pregnant during CHOLBAM treatment are encouraged to enroll. Patients or their health care provider should call Mirum Medical Information 1-855-676-4968. Risk Summary No studies in pregnant women or animal reproduction studies have been conducted with CHOLBAM. Limited published case reports discuss pregnancies in women taking cholic acid for 3β-HSD deficiency resulting in healthy infants. These reports may not adequately inform the presence or absence of drug-associated risk with the use of CHOLBAM during pregnancy. The background risk of major birth defects and miscarriage for the indicated population is unknown. However, the background risk in the U.S. general population of major birth defects is 2-4% and of miscarriage is 15-20% of clinically recognized pregnancies."],"overdosage":["10 OVERDOSAGE Concurrent elevations of serum GGT and ALT may indicate CHOLBAM overdose. If an overdose is suspected, discontinue CHOLBAM and treat symptoms. Continue to monitor laboratory parameters of liver function and consider restarting at a lower dose when the parameters return to baseline [see Dosage and Administration ( 2.2 ) and Warnings and Precautions ( 5.1 )] ."],"description":["11 DESCRIPTION Cholic acid is a bile acid. The chemical formula is C 24 H 40 O 5 , the molecular weight is 408.57 and the chemical structure is: Cholic acid is a white to off-white powder. It is practically insoluble in water and in 0.1 M HCl at 20°C and is sparingly soluble in 0.1 M NaOH at 20°C. It is soluble in glacial acetic acid, alcohols, and acetone. A saturated solution in water at 20°C has a pH of 4.4. CHOLBAM capsules contain 50 mg or 250 mg of cholic acid as the active ingredient in size 2 Swedish orange or size 0 white opaque gelatin capsules, respectively. Inactive ingredients in CHOLBAM include crospovidone, magnesium stearate, and silicified microcrystalline cellulose. The size 2 capsule shells contain gelatin, red iron oxide and titanium dioxide, and the size 0 capsule shells contain gelatin and titanium dioxide. CHOLBAM is administered orally. Cholic acid chemical structure"],"how_supplied":["16 HOW SUPPLIED/STORAGE AND HANDLING 50 mg Capsules CHOLBAM capsules are available as two-piece gelatin capsules with a Swedish orange cap imprinted with “50mg” and Swedish orange body imprinted with “ASK001”. The capsules contain a white or off-white powder and are supplied in bottles of: • 90 capsules (NDC 79378-001-02) 250 mg Capsules CHOLBAM capsules are available as two-piece gelatin capsules with a white cap imprinted with “250mg” and white body imprinted with “ASK002”. The capsules contain a white or off-white powder and are supplied in bottles of: • 90 capsules (NDC 79378-002-02) Storage and Handling Store at 20°C–25 ○ C (68°F–77 ○ F), excursions permitted between 15°C–30 ○ C (59°F–86 ○ F) [see USP Controlled Room Temperature]."],"geriatric_use":["8.5 Geriatric Use Clinical studies of CHOLBAM did not include any patients aged 65 years and over. It is not known if elderly patients respond differently from younger patients."],"pediatric_use":["8.4 Pediatric Use The safety and effectiveness of CHOLBAM have been established in pediatric patients 3 weeks of age and older for the treatment of bile acid synthesis disorders due to SEDs and for adjunctive treatment of patients with PDs including Zellweger spectrum disorders who exhibit manifestations of liver disease, steatorrhea, or complications from decreased fat-soluble vitamin absorption [see Clinical Studies ( 14 )] ."],"effective_time":"20260126","clinical_studies":["14 CLINICAL STUDIES 14.1 Bile Acid Synthesis Disorders due to Single Enzyme Defects The effectiveness of CHOLBAM at dosages of 10 to 15 mg/kg per day in patients with SEDs was assessed in: • Trial 1: a non-randomized, open-label, single-arm trial in 50 patients over an 18‑year period • Trial 2: an extension trial of 12 new patients along with 21 patients who rolled over from Trial 1 (n=33 total). Efficacy data are available for 21 months of treatment. • A published case series of 15 patients with SEDs and 3 patients with PDs. Enrollment criteria in Trials 1 and 2 were based on abnormal urinary bile acid by Fast Atom Bombardment ionization-Mass Spectrometry (FAB-MS). Pre- and post-treatment liver biopsies were performed in a limited number of patients. Documentation of adherence to treatment, concomitant medications, and response to treatment were incomplete in Trial 1. Additional interventions in some patients included supplementation with fat-soluble vitamins, as dictated by the patient’s clinical signs and symptoms. Trials 1 and 2 On average, patients were 4 years of age at the start of cholic acid treatment (range three weeks to 36 years). The majority of patients were treated for an average of 310 weeks (6 years). Patient ages at the end of treatment ranged from 19 to 36 years. These trials were carried out over many years, and data are not available on all patients. Thirty-nine patients in Trial 1 and 5 new patients in Trial 2 received at least one dose of CHOLBAM and had sufficient data available to assess baseline liver function and effects of CHOLBAM treatment. A responder analysis was performed to determine the response to treatment with CHOLBAM. Response to CHOLBAM treatment was assessed by the following laboratory criteria: (1) ALT or AST values reduced to less than 50 U/L, or baseline levels reduced by 80%; (2) total bilirubin values reduced to less than or equal to 1 mg/dL; and (3) no evidence of cholestasis on liver biopsy; and the following clinical criteria: (1) body weight increased by 10% or stable at greater than the 50 th percentile; and (2) survival for greater than 3 years on treatment or alive at the end of Trial 2. CHOLBAM responders were defined as patients who either: (1) met at least two laboratory criteria and were alive at the last follow-up; or (2) met at least one laboratory criteria, had increased body weight, and were alive at the last follow-up. Overall, 28 of 44 patients (64%) were responders. The breakdown by defect type is as follows: Table 4: Response to CHOLBAM Treatment by Type of Single Enzyme Defect *N/A indicates no evaluable patients in the defect subgroup are represented. Single Enzyme Defect Responders/Number Treated (%) 3β-HSD 22/37 (59%) AKR1D1 3/4 (75%) CTX 2/2 (100%) AMACR 1/1 (100%) CYP7A1 N/A* Smith-Lemli-Opitz N/A* Among SED responsive patients, 45% of the responders met the two clinical criteria plus 1 to 3 laboratory criteria and 55% met the weight criteria. Only six patients had pre- and post-treatment liver biopsies in Trial 1. Where biopsies were available, pre-treatment biopsies showed varying degrees of inflammation, bridging fibrosis, and giant cell formation. Post-treatment biopsies generally showed reduced or absent inflammation and reduced or absent giant cell formation. Fibrosis remained but did not progress. It is difficult to evaluate long term survival in patients with SEDs since there is little natural history survival data for comparison. Overall, 41 of 62 (67%) patients with SEDs survived greater than 3 years from trial entry. Thirteen of these 41 patients (32%) survived for 10 to 24 years on treatment. Four patients in Trial 1 underwent liver transplant, including two patients diagnosed with AKR1D1 deficiency, one with 3β-HSD deficiency, and one with CYP7A1 deficiency and two patients in Trial 2, both with AKR1D1. CHOLBAM’s effects on extrahepatic manifestations of SEDs, such as neurologic symptoms have not been established. Case Series A published report of a case series described 15 patients with SEDs; thirteen were diagnosed with 3β-HSD deficiency and two with AKR1D1 deficiency by mass spectrometry and gene sequencing. All patients were treated with cholic acid with a median duration of treatment of 12.4 years (range 5.6 to 15 years). Therapy started at a median age of 3.9 years (range 0.3 to 13.1 years). The mean dose at the start of cholic acid treatment was 13 mg/kg and the mean dose at last follow up was 6 mg/kg. Eight patients were initially treated with oral ursodeoxycholic acid prior to receiving a diagnosis of bile acid synthesis defect, after which they were switched to cholic acid. Initial signs and symptoms included jaundice, hepatosplenomegaly, steatorrhea, or symptoms related to deficiency of a fat-soluble vitamin (K, D or E). Of the 8 patients who received ursodeoxycholic acid initially, the six with 3β-HSD deficiency demonstrated mild clinical improvement. Following treatment with cholic acid, all patients experienced resolution of their pre-existing jaundice and steatorrhea, and all but one experienced resolution of hepatosplenomegaly. Weight and height improved, and sexual maturation progressed normally in all patients. Liver biopsies were performed in 14 patients after at least 5 years of cholic acid treatment and all showed resolution of cholestasis. In one patient with 3β‑HSD deficiency, biliary bile acid analysis while on cholic acid therapy showed enrichment of the bile with cholic acid. 14.2 Peroxisomal Disorders Including Zellweger Spectrum Disorders The effectiveness of CHOLBAM at a dosage of 10 to 15 mg/kg per day in patients with PDs including Zellweger spectrum disorders was assessed in patients in the same trials described in section 14.1 . • Trial 1 treated 29 patients with PDs over an 18‑year period. • Trial 2 treated 2 new patients along with 10 patients who rolled over from Trial 1 (n=12 total). Efficacy data are available from Trial 2 for 21 months of treatment. • Additional efficacy data were obtained from published case reports of 3 patients. Enrollment criteria in Trials 1 and 2 were based on abnormal urinary bile acids analysis by FAB‑MS and a neurologic exam. Most patients received concomitant DHA (docosahexaenoic acid) and Vitamins A, D, E, and K. Documentation of adherence to treatment, concomitant medications, and response to treatment were incomplete during Trial 1. Trials 1 and 2 The majority of patients (80%, 25/31) were less than 2 years of age at the start of CHOLBAM treatment (range 3 weeks to 10 years). The majority of patients were treated for an average of 254 weeks (4.8 years). Sufficient data were available to assess baseline liver function and effects of CHOLBAM treatment in 23 patients in Trial 1 and in one new patient in Trial 2. A responder analysis was performed in the patients who had received at least one dose of CHOLBAM and had sufficient data available to assess baseline liver impairment. Response to CHOLBAM treatment was assessed by the following laboratory criteria: (1) ALT or AST values reduced to less than 50 U/L, or baseline levels reduced by 80%; (2) total bilirubin values reduced to less than or equal to 1 mg/dL; and (3) no evidence of cholestasis on liver biopsy; and the following clinical criteria: (1) body weight increased by 10% or stable at greater than the 50th percentile; and (2) survival for greater than 3 years on treatment or alive at the end of Trial 2 CHOLBAM responders were defined as patients who either: (1) met at least two laboratory criteria and were alive at the last follow-up; or (2) met at least one laboratory criterion, had increased body weight and were alive at the last follow-up. Overall, 11 of 24 patients (46%) were responders. The breakdown by disorder is as follows: Table 5: Response to CHOLBAM Treatment by Type of Peroxisomal Disorders including Zellweger Spectrum Disorders Peroxisomal Disorder Responders/Number Treated (%) Neonatal Adrenoleukodystrophy 3/6 (50%) Generalized Peroxisomal Disorder 1/1 (100%) Refsum Disease 3/4 (75%) Zellweger Syndrome 3/8 (38%) Peroxisomal Disorder, Type Unknown 1/5 (20%) Among responsive patients with PDs, 38% of the responders met the two clinical criteria plus 1 to 3 laboratory criteria and 63% met the weight criteria. There were no PD patients that underwent liver transplant. No evidence of improvement in survival over that seen in historical controls could be demonstrated from the data presented. Overall, 13 of 31 patients (42%) survived greater than 3 years from the time of trial entry. Eight of these 13 patients (62%) survived 10 to 17 years on treatment. Nine patients had both pre- and post-treatment liver biopsies. One patient showed improvement in histology, while the majority of patients remained unchanged. Two patients demonstrated worsening histology, which was consistent with a worsening of other liver laboratory parameters (bilirubin, serum transaminase values). CHOLBAM’s effects on extrahepatic manifestations of PDs including Zellweger spectrum disorders, such as neurologic symptoms have not been established. One patient, who did not have cholestasis on pre-treatment liver biopsy, developed cholestasis on treatment with CHOLBAM and subsequently died. Case Reports In case reports from the literature, a 6-month-old patient with Zellweger syndrome treated with a combination of cholic and chenodeoxycholic acids experienced normalization of serum transaminases and bilirubin, improvement in liver histology, reduced serum and urinary atypical bile acid intermediates, and improvement in steatorrhea and growth. Two patients with Zellweger syndrome treated with oral bile acids showed decreased serum transaminases."],"pharmacokinetics":["12.3 Pharmacokinetics Orally administered cholic acid is subject to the same metabolic pathway as endogenous cholic acid. Cholic acid is absorbed by passive diffusion along the length of the gastrointestinal tract. Once absorbed, cholic acid enters into the body’s bile acid pool and undergoes enterohepatic circulation mainly in conjugated forms. In the liver, cholic acid is conjugated with glycine or taurine by bile acid-CoA synthetase and bile acid-CoA:amino acid N-acyltransferase. Conjugated cholic acid is actively secreted into bile by the BSEP, and then released into the small intestines, along with other components of bile. Conjugated cholic acid is mostly re-absorbed in the ileum mainly by the apical-sodium-dependent-bile acid transporter, passed back to the liver by transporters including sodium-taurocholate cotransporting polypeptide and organic anion transport protein and enters another cycle of enterohepatic circulation. Any conjugated cholic acid not absorbed in the ileum passes into the colon where deconjugation and 7-dehydroxylation are mediated by bacteria to form cholic acid and deoxycholic acid, which may be re-absorbed in the colon or excreted in the feces. The loss of cholic acid is compensated by de-novo synthesis of cholic acids from cholesterol to maintain the bile acid pool in healthy subjects."],"adverse_reactions":["6 ADVERSE REACTIONS The following clinically significant adverse reaction is described elsewhere in the labeling: • Exacerbation of Liver Impairment [see Warnings and Precautions ( 5.1 )] Most common adverse reactions (≥1%) are diarrhea, reflux esophagitis, malaise, jaundice, skin lesion, nausea, abdominal pain, intestinal polyp, urinary tract infection, and peripheral neuropathy. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Mirum Pharmaceuticals at 1-855-MRM-4YOU or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. Clinical safety experience with CHOLBAM consists of: • Trial 1: a non-randomized, open-label, single-arm trial of 50 patients with bile acid synthesis disorders due to SEDs and 29 patients with PDs including Zellweger spectrum disorders. Safety data are available over the 18 years of the trial. • Trial 2: an extension trial of 12 new patients (10 SED and 2 PD) along with 31 (21 SED and 10 PD) patients who rolled over from Trial 1. Safety data are available for 3 years and 11 months of treatment. Adverse events were not collected systematically in either of these trials. Most patients received an oral dose of 10 to 15 mg/kg/day of CHOLBAM. Deaths In Trial 1, among the 50 patients with SEDs, 5 patients aged 1 year or less died, which included three patients originally diagnosed with AKR1D1 deficiency, one with 3β-HSD deficiency and one with CYP7A1 deficiency. The cause of death was attributed to progression of underlying liver disease in every patient. Of 29 patients in Trial 1 with PDs including Zellweger spectrum disorders, 12 patients between the ages of 7 months and 2.5 years died. In the majority of these patients (8/12), the cause of death was attributed to progression of underlying liver disease or to a worsening of their primary illness. Two additional patients in Trial 1 (1 SED and 1 PD) died who had been off study medication for more than one year with the cause of death most likely being a progression of underlying liver disease. Of the patients who died with disease progression, laboratory testing showed abnormal serum transaminases, bilirubin, or cholestasis on liver biopsy suggesting worsening of their underlying cholestasis. In Trial 2, among the 31 patients with SED, two patients (1 new patient and 1 who rolled over from Trial 1) died. The cause of death in both cases was unrelated to their primary treatment or progression of their underlying liver disease. Of the 12 patients with PD in Trial 2, four patients died between the ages of 4 and 8 years (1 new patient and 3 who rolled over from Trial 1). The cause of death in three of these patients was attributed to progression of underlying liver disease or to a worsening of their primary illness. Worsening of Liver Impairment Seven patients in Trial 1 (4 SED and 3 PD) and 3 patients in Trial 2 (1 SED and 2 PD) experienced worsening serum transaminases, elevated bilirubin values, or worsening cholestasis on liver biopsy during treatment [see Warnings and Precautions ( 5.1 )] . Common Adverse Reactions There were 12 adverse reactions reported across 9 patients in the trials, with diarrhea being the most common reaction in approximately 2% of the patient population. All other adverse reactions represented 1% of the patient population. The breakdown by trial follows: Table 3: Most Common Adverse Reactions in Trials 1 and 2 *Adverse reactions that occurred in new patients Adverse Reactions Trial 1 Trial 2* Overall n (%) Diarrhea 1 2* 3 (2) Reflux Esophagitis 1 0 1 (1) Malaise 1 0 1 (1) Jaundice 1 0 1 (1) Skin lesion 1 0 1 (1) Nausea 0 1* 1 (1) Abdominal Pain 0 1* 1 (1) Intestinal Polyp 0 1* 1 (1) Urinary Tract Infection 0 1* 1 (1) Peripheral Neuropathy 0 1 1 (1) Only one of the reactions (peripheral neuropathy) resulted in discontinuation of medication for a patient in Trial 2. An additional five SED patients (3 from Trial 1 and 2 from Trial 2) and 1 PD patient (Trial 1) discontinued medication and withdrew from the study due to a worsening of their primary disease. The development of symptomatic cholelithiasis requiring cholecystectomy has been reported in a single patient with 3β-HSD deficiency. 6.2. Postmarketing Experience The following adverse reactions have been identified during post-approval use of CHOLBAM. Because these reactions are reported voluntarily from a population of uncertain size, it is not always possible to reliably estimate their relative frequency or to establish a causal relationship to CHOLBAM exposure: • Gastrointestinal disorders : discomfort and distention, emesis, constipation • General disorders and administrative site conditions : pyrexia/fever • Skin and subcutaneous tissue disorders : rash"],"contraindications":["4 CONTRAINDICATIONS None. None ( 4 )"],"drug_interactions":["7 DRUG INTERACTIONS • Bile Salt Efflux Pump (BSEP) Inhibitors (e.g., cyclosporine): Avoid concomitant use; if concomitant use is necessary, monitor serum transaminases and bilirubin ( 7.1 ) • Bile Acid Resins and Aluminum-Based Antacids: Take CHOLBAM at least 1 hour before or 4 to 6 hours (or at as great an interval as possible) after a bile acid binding resin or aluminum-based antacids. ( 2.3 , 7.1 ) 7.1. Effects of Other Drugs on CHOLBAM Drug interactions with CHOLBAM mainly relate to agents capable of interrupting the enterohepatic circulation of bile acids. Inhibitors of Bile Acid Transporters Avoid concomitant use of inhibitors of the bile salt efflux pump (BSEP) such as cyclosporine. Concomitant medications that inhibit canalicular membrane bile acid transporters such as the BSEP may exacerbate accumulation of conjugated bile salts in the liver and result in clinical symptoms. If concomitant use is deemed necessary, monitoring of serum transaminases and bilirubin is recommended. Bile Acid Binding Resins Bile acid binding resins such as cholestyramine, colestipol, or colesevelam adsorb and reduce bile acid absorption and may reduce the efficacy of CHOLBAM. Take CHOLBAM at least 1 hour before or 4 to 6 hours (or at as great an interval as possible) after a bile acid binding resin [see Dosage and Administration ( 2.3 )] . Aluminum-Based Antacids Aluminum-based antacids have been shown to adsorb bile acids in vitro and can reduce the availability of CHOLBAM. Take CHOLBAM at least 1 hour before or 4 to 6 hours (or at as great an interval as possible) after an aluminum-based antacid [see Dosage and Administration ( 2.3 )] ."],"mechanism_of_action":["12.1 Mechanism of Action Cholic acid is a primary bile acid synthesized from cholesterol in the liver. In bile acid synthesis disorders due to SEDs in the biosynthetic pathway, and in PDs including Zellweger spectrum disorders, deficiency of primary bile acids leads to unregulated accumulation of intermediate bile acids and cholestasis. Bile acids facilitate fat digestion and absorption by forming mixed micelles and facilitate absorption of fat-soluble vitamins in the intestine. Endogenous bile acids including cholic acid enhance bile flow and provide the physiologic feedback inhibition of bile acid synthesis. The mechanism of action of cholic acid has not been fully established; however, it is known that cholic acid and its conjugates are endogenous ligands of the nuclear receptor, farnesoid X receptor (FXR). FXR regulates enzymes and transporters that are involved in bile acid synthesis and in the enterohepatic circulation to maintain bile acid homeostasis under normal physiologic conditions."],"clinical_pharmacology":["12 CLINICAL PHARMACOLOGY 12.1 Mechanism of Action Cholic acid is a primary bile acid synthesized from cholesterol in the liver. In bile acid synthesis disorders due to SEDs in the biosynthetic pathway, and in PDs including Zellweger spectrum disorders, deficiency of primary bile acids leads to unregulated accumulation of intermediate bile acids and cholestasis. Bile acids facilitate fat digestion and absorption by forming mixed micelles and facilitate absorption of fat-soluble vitamins in the intestine. Endogenous bile acids including cholic acid enhance bile flow and provide the physiologic feedback inhibition of bile acid synthesis. The mechanism of action of cholic acid has not been fully established; however, it is known that cholic acid and its conjugates are endogenous ligands of the nuclear receptor, farnesoid X receptor (FXR). FXR regulates enzymes and transporters that are involved in bile acid synthesis and in the enterohepatic circulation to maintain bile acid homeostasis under normal physiologic conditions. 12.3 Pharmacokinetics Orally administered cholic acid is subject to the same metabolic pathway as endogenous cholic acid. Cholic acid is absorbed by passive diffusion along the length of the gastrointestinal tract. Once absorbed, cholic acid enters into the body’s bile acid pool and undergoes enterohepatic circulation mainly in conjugated forms. In the liver, cholic acid is conjugated with glycine or taurine by bile acid-CoA synthetase and bile acid-CoA:amino acid N-acyltransferase. Conjugated cholic acid is actively secreted into bile by the BSEP, and then released into the small intestines, along with other components of bile. Conjugated cholic acid is mostly re-absorbed in the ileum mainly by the apical-sodium-dependent-bile acid transporter, passed back to the liver by transporters including sodium-taurocholate cotransporting polypeptide and organic anion transport protein and enters another cycle of enterohepatic circulation. Any conjugated cholic acid not absorbed in the ileum passes into the colon where deconjugation and 7-dehydroxylation are mediated by bacteria to form cholic acid and deoxycholic acid, which may be re-absorbed in the colon or excreted in the feces. The loss of cholic acid is compensated by de-novo synthesis of cholic acids from cholesterol to maintain the bile acid pool in healthy subjects."],"indications_and_usage":["1 INDICATIONS AND USAGE CHOLBAM is a bile acid indicated for: • Treatment of bile acid synthesis disorders due to single enzyme defects (SEDs). ( 1.1 ) • Adjunctive treatment of peroxisomal disorders (PDs) including Zellweger spectrum disorders in patients who exhibit manifestations of liver disease, steatorrhea or complications from decreased fat-soluble vitamin absorption. ( 1.2 ) Limitations of use: The safety and effectiveness of CHOLBAM on extrahepatic manifestations of bile acid synthesis disorders due to SEDs or PDs including Zellweger spectrum disorders have not been established. ( 1.3 ). 1.1 Bile Acid Synthesis Disorders Due to Single Enzyme Defects CHOLBAM is indicated for the treatment of bile acid synthesis disorders due to single enzyme defects (SEDs). 1.2. Peroxisomal Disorders Including Zellweger Spectrum Disorders CHOLBAM is indicated for adjunctive treatment of peroxisomal disorders (PDs) including Zellweger spectrum disorders in patients who exhibit manifestations of liver disease, steatorrhea or complications from decreased fat-soluble vitamin absorption. 1.3. Limitations of Use The safety and effectiveness of CHOLBAM on extrahepatic manifestations of bile acid synthesis disorders due to SEDs or PDs including Zellweger spectrum disorders have not been established."],"warnings_and_cautions":["5 WARNINGS AND PRECAUTIONS Exacerbation of Liver Impairment: Monitor liver function. Discontinue CHOLBAM if liver function worsens while on treatment. ( 5.1 ) 5.1 Exacerbation of Liver Impairment In clinical trials, evidence of liver impairment was present before treatment with CHOLBAM in approximately 86% (44/51) of patients with bile acid synthesis disorders due to SEDs and in approximately 50% (14/28) of patients with PDs including Zellweger spectrum disorders. Five of the patients (3 SED and 2 PD) with liver impairment at baseline experienced worsening serum transaminases, elevated bilirubin values, or worsening cholestasis on liver biopsy following treatment. Five additional patients (2 SED and 3 PD) who did not have baseline cholestasis experienced exacerbation of their liver disease while on treatment. In patients with cirrhosis, cases of severe hepatotoxicity have also been observed following postmarket use of CHOLBAM. Exacerbation of liver impairment by CHOLBAM in these patients cannot be ruled out. Six patients with SEDs underwent liver transplant, including four patients diagnosed with AKR1D1 deficiency, one with 3β-HSD deficiency, and one with CYP7A1 deficiency. Concurrent elevations of serum GGT and ALT may indicate CHOLBAM overdose [see Dosage and Administration ( 2.2 ) and Overdosage ( 10 )] . Monitor liver function and discontinue CHOLBAM in patients who develop worsening of liver function while on treatment. Discontinue treatment with CHOLBAM at any time if there are clinical or laboratory indicators of worsening liver function or cholestasis [see Dosage and Administration ( 2.2 )]."],"clinical_studies_table":["<table styleCode=\"Noautorules\" width=\"100%\"><caption>Table 4: Response to CHOLBAM Treatment by Type of Single Enzyme Defect</caption><col width=\"42%\"/><col width=\"42%\"/><tfoot><tr><td align=\"left\" colspan=\"2\" valign=\"top\">*N/A indicates no evaluable patients in the defect subgroup are represented.</td></tr></tfoot><tbody><tr><td align=\"center\" styleCode=\"Rrule Botrule Lrule Toprule \" valign=\"middle\"><paragraph><content styleCode=\"bold\">Single Enzyme Defect</content></paragraph></td><td align=\"center\" styleCode=\"Rrule Botrule Lrule Toprule \" valign=\"middle\"><paragraph><content styleCode=\"bold\">Responders/Number Treated (%)</content></paragraph></td></tr><tr><td align=\"center\" styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>3&#x3B2;-HSD</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>22/37 (59%)</paragraph></td></tr><tr><td align=\"center\" styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>AKR1D1</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>3/4 (75%)</paragraph></td></tr><tr><td align=\"center\" styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>CTX</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>2/2 (100%)</paragraph></td></tr><tr><td align=\"center\" styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>AMACR</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>1/1 (100%)</paragraph></td></tr><tr><td align=\"center\" styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>CYP7A1</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>N/A*</paragraph></td></tr><tr><td align=\"center\" styleCode=\"Rrule Botrule Lrule Toprule \" valign=\"top\"><paragraph>Smith-Lemli-Opitz</paragraph></td><td align=\"center\" styleCode=\"Rrule Botrule Lrule Toprule \" valign=\"top\"><paragraph>N/A*</paragraph></td></tr></tbody></table>","<table width=\"100%\"><caption>Table 5: Response to CHOLBAM Treatment by Type of Peroxisomal Disorders including Zellweger Spectrum Disorders</caption><col width=\"38%\"/><col width=\"38%\"/><thead><tr><th align=\"left\" styleCode=\"Rrule Botrule Lrule Toprule \" valign=\"top\"><content styleCode=\"bold\">Peroxisomal Disorder</content></th><th align=\"center\" styleCode=\"Rrule Botrule Lrule Toprule \" valign=\"top\"><content styleCode=\"bold\">Responders/Number Treated (%)</content></th></tr></thead><tbody><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>Neonatal Adrenoleukodystrophy</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"middle\"><paragraph>3/6 (50%)</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>Generalized Peroxisomal Disorder</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"middle\"><paragraph>1/1 (100%)</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>Refsum Disease</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"middle\"><paragraph>3/4 (75%)</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>Zellweger Syndrome</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"middle\"><paragraph>3/8 (38%)</paragraph></td></tr><tr><td styleCode=\"Rrule Botrule Lrule Toprule \" valign=\"top\"><paragraph>Peroxisomal Disorder, Type Unknown</paragraph></td><td align=\"center\" styleCode=\"Rrule Botrule Lrule Toprule \" valign=\"middle\"><paragraph>1/5 (20%)</paragraph></td></tr></tbody></table>"],"nonclinical_toxicology":["13 NONCLINICAL TOXICOLOGY 13.1 Carcinogenesis, Mutagenesis, Impairment of Fertility Carcinogenicity, genetic toxicology, and nonclinical fertility studies have not been performed with cholic acid. 13.2 Animal Toxicology and/or Pharmacology In the PEX2 -/- mouse model of peroxisomal disorders, feeding with a combination of cholic acid and ursodeoxycholic acid normalized C24 bile acid concentrations in bile to that of untreated control animals. Although growth was only mildly improved, there was near complete normalization of stool fat content, resolution of steatorrhea, and improved survival. Bile acid feeding reduced the number of cholestatic deposits in bile ducts and alleviated cholangitis but exacerbated the degree of hepatic steatosis and mitochondrial and cellular damage in the peroxisome-deficient livers of these animals."],"adverse_reactions_table":["<table styleCode=\"Noautorules\" width=\"100%\"><caption>Table 3: Most Common Adverse Reactions in Trials 1 and 2</caption><col width=\"25%\"/><col width=\"25%\"/><col width=\"25%\"/><col width=\"25%\"/><tfoot><tr><td align=\"left\" colspan=\"4\" valign=\"top\">*Adverse reactions that occurred in new patients</td></tr></tfoot><tbody><tr><td styleCode=\"Rrule Botrule Lrule Toprule \" valign=\"bottom\"><paragraph><content styleCode=\"bold\">Adverse Reactions</content></paragraph></td><td align=\"center\" styleCode=\"Rrule Botrule Lrule Toprule \" valign=\"bottom\"><paragraph><content styleCode=\"bold\">Trial 1</content></paragraph></td><td align=\"center\" styleCode=\"Rrule Botrule Lrule Toprule \" valign=\"bottom\"><paragraph><content styleCode=\"bold\">Trial 2*</content></paragraph></td><td align=\"center\" styleCode=\"Rrule Botrule Lrule Toprule \" valign=\"bottom\"><paragraph><content styleCode=\"bold\">Overall n (%)</content></paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>Diarrhea</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>1</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>2*</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>3 (2)</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>Reflux Esophagitis</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>1</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>0</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>1 (1)</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>Malaise</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>1</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>0</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>1 (1)</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>Jaundice</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>1</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>0</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>1 (1)</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>Skin lesion</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>1</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>0</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>1 (1)</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>Nausea</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>0</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>1*</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>1 (1)</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>Abdominal Pain</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>0</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>1*</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>1 (1)</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>Intestinal Polyp</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>0</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>1*</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>1 (1)</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>Urinary Tract Infection</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>0</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>1*</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>1 (1)</paragraph></td></tr><tr><td styleCode=\"Rrule Botrule Lrule Toprule \" valign=\"top\"><paragraph>Peripheral Neuropathy</paragraph></td><td align=\"center\" styleCode=\"Rrule Botrule Lrule Toprule \" valign=\"top\"><paragraph>0</paragraph></td><td align=\"center\" styleCode=\"Rrule Botrule Lrule Toprule \" valign=\"top\"><paragraph>1</paragraph></td><td align=\"center\" styleCode=\"Rrule Botrule Lrule Toprule \" valign=\"top\"><paragraph>1 (1)</paragraph></td></tr></tbody></table>"],"information_for_patients":["17 PATIENT COUNSELING INFORMATION Exacerbation of Liver Impairment [see Warnings and Precautions ( 5.1 )] • Advise patients that they will need to undergo laboratory testing periodically while on treatment to assess liver function. • Advise patients that CHOLBAM may worsen liver impairment and that they should immediately report to their health care provider any symptoms associated with liver impairment (e.g., yellowing of the skin or the whites of the eye, dark or tea-colored urine, pain on the right side of stomach, bleeding or bruising occurs more easily than normal, or increased lethargy) Administration [see Dosage and Administration ( 2.3 ) ] Advise patients: • to take CHOLBAM with food. • to take CHOLBAM at least one hour before or 4 to 6 hours after taking a bile acid binding resin or an aluminum-based antacid. • not to crush or chew the capsules. • for infants and children who cannot swallow capsules, the capsules can be opened and the contents mixed with either infant formula or expressed breast milk (for younger children), or soft food such as mashed potatoes or apple puree (for older children and adults) in order to mask any unpleasant taste: 1. Hold the capsule over the prepared liquid/food, gently twist open, and allow the contents to fall into the liquid/food. 2. Mix the entire capsule contents with one or two tablespoonfuls (15 mL or 30 mL) of infant formula, expressed breast milk, or soft food such as mashed potatoes or apple puree. 3. Stir for 30 seconds. 4. The capsule contents will remain as fine granules in the milk or food and will not dissolve. 5. Administer the mixture immediately. Pregnancy Surveillance Program [see Use in Specific Populations ( 8.1 )] Inform patients about the pregnancy surveillance program that monitors pregnancy outcomes in women exposed to CHOLBAM during pregnancy. Manufactured by: Patheon France S.A.S. 38300 Bourgoin-Jallieu, France Manufactured for: Mirum Pharmaceuticals, Inc. Foster City, CA 94404 © 2026 Mirum Pharmaceuticals, Inc. CHOLBAM ® is a registered trademark of Mirum Pharmaceuticals, Inc. LB00132v2"],"dosage_and_administration":["2 DOSAGE AND ADMINISTRATION • The recommended dosage is 10 to 15 mg/kg once daily or in two divided doses, in pediatric patients and adults. See prescribing information for weight-based dosing tables. ( 2.1 ) • The recommended dosage in patients with concomitant familial hypertriglyceridemia is 11 to 17 mg/kg once daily or in two divided doses and is adjusted based on clinical response. ( 2.1 ) • Monitor AST, ALT, GGT, alkaline phosphatase, bilirubin and INR every month for the first 3 months, every 3 months for the next 9 months, every 6 months during the next three years and annually thereafter. Administer the lowest dose that effectively maintains liver function. ( 2.2 ) • Discontinue CHOLBAM if liver function does not improve within 3 months of starting treatment, if complete biliary obstruction develops, or if there are persistent clinical or laboratory indicators of worsening liver function or cholestasis; continue to monitor liver function and consider restarting at a lower dose when parameters return to baseline. ( 2.2 , 5.1 ) Administration Instructions: • Take with food. ( 2.3 ) • Do not crush or chew the capsules. For patients unable to swallow the capsules, the capsules can be opened and the contents mixed with drink/food ( 2.3 ) 2.1. Dosage Regimen for Bile Acid Synthesis Disorders Due to SEDs and PDs Including Zellweger Spectrum Disorders The recommended dosage of CHOLBAM is 10 to 15 mg/kg administered orally once daily or in two divided doses, in pediatric patients and adults. Table 1 and Table 2 show the number of capsules that should be administered daily to approximate a dosage of 10 mg/kg/day and 15 mg/kg/day, respectively, using the available 50 mg and 250 mg capsules alone or in combination. Table 1: Number of CHOLBAM Capsules Needed to Achieve a Recommended Dosage of 10 mg/kg/day 10 mg/kg/day Dosage Body Weight (kg) Number of 50 mg capsules Number of 250 mg capsules 4 to 6 1 0 7 to 10 2 0 11 to 15 3 0 16 to 20 4 0 21 to 25 0 1 26 to 30 1 1 31 to 35 2 1 36 to 40 3 1 41 to 45 4 1 46 to 50 0 2 51 to 55 1 2 56 to 60 2 2 61 to 65 3 2 66 to 70 4 2 71 to 75 0 3 76 to 80 1 3 Table 2: Number of CHOLBAM Capsules Needed to Achieve a Recommended Dosage of 15 mg/kg/day 15 mg/kg/day Dosage Body Weight (kg) Number of 50 mg capsules Number of 250 mg capsules 4 to 5 1 0 6 to 9 2 0 10 to 13 3 0 14 to 16 4 0 17 to 19 0 1 20 to 23 1 1 24 to 26 2 1 27 to 29 3 1 30 to 33 4 1 34 to 36 0 2 37 to 39 1 2 40 to 43 2 2 44 to 46 3 2 47 to 49 4 2 50 to 53 0 3 54 to 56 1 3 57 to 59 2 3 60 to 63 3 3 64 to 66 4 3 67 to 69 0 4 70 to 73 1 4 74 to 76 2 4 77 to 79 3 4 80 4 4 Patients with newly diagnosed, or a family history of, familial hypertriglyceridemia may have poor absorption of CHOLBAM from the intestine and require a 10% increase in the recommended dosage to account for losses due to malabsorption. The recommended dosage of CHOLBAM in patients with concomitant familial hypertriglyceridemia is 11 to 17 mg/kg orally once daily, or in two divided doses. Adequacy of the dosage regimen can be determined by monitoring the patient’s clinical response including steatorrhea and laboratory values of serum transaminases, bilirubin, and prothrombin time/international normalized ratio (PT/INR). 2.2. Treatment Monitoring Treatment with CHOLBAM should be initiated and monitored by an experienced hepatologist or pediatric gastroenterologist. Monitor serum aspartate aminotransferase (AST), serum alanine aminotransferase (ALT), serum gamma glutamyltransferase (GGT), alkaline phosphatase, bilirubin, and INR every month for the first 3 months, every 3 months for the next 9 months, every 6 months during the subsequent three years, and annually thereafter. Monitor more frequently during periods of rapid growth, concomitant disease, and pregnancy. Administer the lowest dose of CHOLBAM that effectively maintains liver function [see Warnings and Precautions ( 5.1 )] . Discontinue treatment with CHOLBAM if liver function does not improve within 3 months of the start of treatment or if complete biliary obstruction develops. Discontinue treatment with CHOLBAM at any time if there are persistent clinical or laboratory indicators of worsening liver function or cholestasis [see Warnings and Precautions ( 5.1 )] . Concurrent elevations of serum GGT and serum ALT may indicate CHOLBAM overdose [see Overdosage ( 10 )] . Continue to monitor laboratory parameters of liver function and consider restarting at a lower dose when parameters return to baseline. Assessment of serum or urinary bile acid levels using mass spectrometry is used in the diagnosis of bile acid synthesis disorders due to SEDs and PDs including Zellweger spectrum disorders. The utility of bile acid measurements in monitoring the clinical course of patients and in decisions regarding dose adjustment has not been demonstrated. 2.3 Administration Instructions • Take CHOLBAM with food. • Take CHOLBAM at least 1 hour before or 4 to 6 hours (or at as great an interval as possible) after a bile acid binding resin or aluminum-based antacid. • Do not crush or chew the capsules. • For patients unable to swallow the capsules, open the capsules and mix the contents with infant formula or expressed breast milk (for younger children), or soft food such as mashed potatoes or apple puree (for older children and adults) in order to mask any unpleasant taste: 1. Hold the capsule over the prepared liquid/food, gently twist open, and allow the contents to fall into the liquid/food. 2. Mix the entire capsule contents with one or two tablespoons (15 mL to 30 mL) of infant formula, expressed breast milk, or soft food such as mashed potatoes or apple puree. 3. Stir for 30 seconds. 4. The capsule contents will remain as fine granules in the milk or food and will not dissolve. 5. Administer the mixture immediately."],"spl_product_data_elements":["CHOLBAM cholic acid CHOLIC ACID CHOLIC ACID MAGNESIUM STEARATE MICROCRYSTALLINE CELLULOSE CROSPOVIDONE, UNSPECIFIED GELATIN, UNSPECIFIED FERRIC OXIDE RED TITANIUM DIOXIDE 50mg;ASK001 CHOLBAM cholic acid CHOLIC ACID CHOLIC ACID MAGNESIUM STEARATE MICROCRYSTALLINE CELLULOSE CROSPOVIDONE, UNSPECIFIED GELATIN, UNSPECIFIED TITANIUM DIOXIDE 250mg;ASK002"],"dosage_forms_and_strengths":["3 DOSAGE FORMS AND STRENGTHS CHOLBAM is available in two capsule strengths: • 50 mg capsule: Size number 2 Swedish orange capsule with cap imprinted with “50mg” and body imprinted with “ASK001”. The capsules contain a white to off-white powder. • 250 mg capsule: Size number 0 white capsule with a cap imprinted with “250mg” and body imprinted with “ASK002”. The capsules contain a white to off-white powder. Capsules: 50 mg, 250 mg ( 3 )"],"use_in_specific_populations":["8 USE IN SPECIFIC POPULATIONS 8.1 Pregnancy Pregnancy Surveillance Program There is a pregnancy surveillance program that monitors pregnancy outcomes in women exposed to CHOLBAM during pregnancy. Women who become pregnant during CHOLBAM treatment are encouraged to enroll. Patients or their health care provider should call Mirum Medical Information 1-855-676-4968. Risk Summary No studies in pregnant women or animal reproduction studies have been conducted with CHOLBAM. Limited published case reports discuss pregnancies in women taking cholic acid for 3β-HSD deficiency resulting in healthy infants. These reports may not adequately inform the presence or absence of drug-associated risk with the use of CHOLBAM during pregnancy. The background risk of major birth defects and miscarriage for the indicated population is unknown. However, the background risk in the U.S. general population of major birth defects is 2-4% and of miscarriage is 15-20% of clinically recognized pregnancies. 8.2 Lactation Risk Summary Endogenous cholic acid is present in human milk. Clinical lactation studies have not been conducted to assess the presence of CHOLBAM in human milk, the effects of CHOLBAM on the breastfed infant, or the effects of CHOLBAM on milk production. There are no animal lactation data and no data from case reports available in the published literature. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for CHOLBAM and any potential adverse effects on the breastfed infant from CHOLBAM or from the underlying maternal condition. 8.4 Pediatric Use The safety and effectiveness of CHOLBAM have been established in pediatric patients 3 weeks of age and older for the treatment of bile acid synthesis disorders due to SEDs and for adjunctive treatment of patients with PDs including Zellweger spectrum disorders who exhibit manifestations of liver disease, steatorrhea, or complications from decreased fat-soluble vitamin absorption [see Clinical Studies ( 14 )] . 8.5 Geriatric Use Clinical studies of CHOLBAM did not include any patients aged 65 years and over. It is not known if elderly patients respond differently from younger patients. 8.6. Hepatic Impairment Discontinue treatment with CHOLBAM if liver function does not improve within 3 months of the start of treatment. Discontinue treatment with CHOLBAM at any time if there are clinical or laboratory indicators of worsening liver function or cholestasis [see Warnings and Precautions ( 5.1 ), Overdosage ( 10 ), and Nonclinical Toxicology ( 13.2 )]. Continue to monitor laboratory parameters of liver function and consider restarting at a lower dose when the parameters return to baseline."],"dosage_and_administration_table":["<table ID=\"_Ref196814973\" cellpadding=\"0pt\" width=\"100%\"><caption>Table 1: Number of CHOLBAM Capsules Needed to Achieve a Recommended Dosage of 10 mg/kg/day</caption><col width=\"40%\"/><col width=\"30%\"/><col width=\"30%\"/><thead><tr><th align=\"left\" styleCode=\"Rrule Botrule Lrule Toprule \" valign=\"top\"/><th align=\"center\" colspan=\"2\" styleCode=\"Rrule Botrule Lrule Toprule \" valign=\"bottom\"><content styleCode=\"bold\">10 mg/kg/day Dosage</content></th></tr><tr><th align=\"left\" styleCode=\"Rrule Lrule Botrule \" valign=\"bottom\"><content styleCode=\"bold\">Body Weight (kg)</content></th><th align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><content styleCode=\"bold\">Number of  50 mg capsules</content></th><th align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><content styleCode=\"bold\">Number of 250 mg capsules</content></th></tr></thead><tbody><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>4 to 6</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>1</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>0</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>7 to 10</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>2</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>0</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>11 to 15</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>3</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>0</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>16 to 20</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>4</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>0</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>21 to 25</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>0</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>1</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>26 to 30</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>1</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>1</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>31 to 35</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>2</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>1</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>36 to 40</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>3</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>1</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>41 to 45</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>4</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>1</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>46 to 50</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>0</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>2</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>51 to 55</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>1</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>2</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>56 to 60</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>2</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>2</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>61 to 65</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>3</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>2</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>66 to 70</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>4</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>2</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>71 to 75</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>0</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>3</paragraph></td></tr><tr><td styleCode=\"Rrule Botrule Lrule Toprule \" valign=\"top\"><paragraph>76 to 80</paragraph></td><td align=\"center\" styleCode=\"Rrule Botrule Lrule Toprule \" valign=\"top\"><paragraph>1</paragraph></td><td align=\"center\" styleCode=\"Rrule Botrule Lrule Toprule \" valign=\"top\"><paragraph>3</paragraph></td></tr></tbody></table>","<table ID=\"_Ref196823364\" cellpadding=\"0pt\" width=\"100%\"><caption>Table 2: Number of CHOLBAM Capsules Needed to Achieve a Recommended Dosage of 15 mg/kg/day</caption><col width=\"40%\"/><col width=\"30%\"/><col width=\"30%\"/><tbody><tr><td styleCode=\"Rrule Botrule Lrule Toprule \" valign=\"top\"/><td align=\"center\" colspan=\"2\" styleCode=\"Rrule Botrule Lrule Toprule \" valign=\"bottom\"><paragraph><content styleCode=\"bold\">15 mg/kg/day Dosage</content></paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"bottom\"><paragraph><content styleCode=\"bold\">Body Weight (kg)</content></paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"bottom\"><paragraph><content styleCode=\"bold\">Number of 50 mg capsules</content></paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"bottom\"><paragraph><content styleCode=\"bold\">Number of 250 mg capsules</content></paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>4 to 5</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>1</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>0</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>6 to 9</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>2</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>0</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>10 to 13</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>3</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>0</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>14 to 16</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>4</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>0</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>17 to 19</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>0</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>1</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>20 to 23</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>1</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>1</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>24 to 26</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>2</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>1</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>27 to 29</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>3</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>1</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>30 to 33</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>4</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>1</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>34 to 36</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>0</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>2</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>37 to 39</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>1</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>2</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>40 to 43</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>2</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>2</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>44 to 46</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>3</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>2</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>47 to 49</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>4</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>2</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>50 to 53</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>0</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>3</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>54 to 56</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>1</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>3</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>57 to 59</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>2</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>3</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>60 to 63</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>3</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>3</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>64 to 66</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>4</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>3</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>67 to 69</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>0</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>4</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>70 to 73</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>1</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>4</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>74 to 76</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>2</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>4</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Botrule \" valign=\"top\"><paragraph>77 to 79</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>3</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"top\"><paragraph>4</paragraph></td></tr><tr><td styleCode=\"Rrule Botrule Lrule Toprule \" valign=\"top\"><paragraph>80</paragraph></td><td align=\"center\" styleCode=\"Rrule Botrule Lrule Toprule \" valign=\"top\"><paragraph>4</paragraph></td><td align=\"center\" styleCode=\"Rrule Botrule Lrule Toprule \" valign=\"top\"><paragraph>4</paragraph></td></tr></tbody></table>"],"animal_pharmacology_and_or_toxicology":["13.2 Animal Toxicology and/or Pharmacology In the PEX2 -/- mouse model of peroxisomal disorders, feeding with a combination of cholic acid and ursodeoxycholic acid normalized C24 bile acid concentrations in bile to that of untreated control animals. Although growth was only mildly improved, there was near complete normalization of stool fat content, resolution of steatorrhea, and improved survival. Bile acid feeding reduced the number of cholestatic deposits in bile ducts and alleviated cholangitis but exacerbated the degree of hepatic steatosis and mitochondrial and cellular damage in the peroxisome-deficient livers of these animals."],"package_label_principal_display_panel":["PACKAGE/LABEL PRINCIPAL DISPLAY PANEL Each capsule contains: Cholic acid..................50 mg DOSAGE: See the package insert for dosage information. WARNING: As with all medications, keep out of the reach of children. Store 20-25°C (68-77°F), excursions permitted between 15-30°C (59-86°F). [see USP Controlled Room Temperature]. Store in the original package in order to protect from light. Mirum Pharmaceuticals, Inc. NDC 79378-001-02 ; 90 capsules; Cholbam® (cholic acid) capsules (cholic acid) capsules; 50 mg; Rx Only Manufactured for: Mirum Pharmaceuticals, Inc. Foster City, CA 94404 Manufactured by: Patheon France SA 38300 Bourgoin-Jallieu, France For product information please call 1-855-MRM-4YOU CHOLBAM-50MG","PACKAGE/LABEL PRINCIPAL DISPLAY PANEL Each capsule contains: Cholic acid..................250 mg DOSAGE: See the package insert for dosage information. WARNING: As with all medications, keep out of the reach of children. Store 20-25°C (68-77°F), excursions permitted between 15-30°C (59-86°F). [see USP Controlled Room Temperature]. Store in the original package in order to protect from light. Mirum Pharmaceuticals, Inc. NDC 79378-002-02 ; 90 capsules; Cholbam® (cholic acid) capsules (cholic acid) capsules; 250 mg; Rx Only Manufactured for: Mirum Pharmaceuticals, Inc. Foster City, CA 94404 Manufactured by: Patheon France SA 38300 Bourgoin-Jallieu, France For product information please call 1-855-MRM-4YOU CHOLBAM-250MG"],"carcinogenesis_and_mutagenesis_and_impairment_of_fertility":["13.1 Carcinogenesis, Mutagenesis, Impairment of Fertility Carcinogenicity, genetic toxicology, and nonclinical fertility studies have not been performed with cholic acid."]},"tags":[{"label":"Bile Acid","category":"class"},{"label":"Small Molecule","category":"modality"},{"label":"G-protein coupled bile acid receptor 1","category":"target"},{"label":"GPBAR1","category":"gene"},{"label":"NR1H4","category":"gene"},{"label":"A05AA03","category":"atc"},{"label":"Oral","category":"route"},{"label":"Capsule","category":"form"},{"label":"Off-Patent","category":"patent"},{"label":"Active","category":"status"},{"label":"3-Beta-hydroxy-delta-5-C27-steroid dehydrogenase deficiency","category":"indication"},{"label":"Calculus in biliary tract","category":"indication"},{"label":"Cholestanol storage disease","category":"indication"},{"label":"Constipation","category":"indication"},{"label":"Delta-4-3-oxosteroid-5-beta-reductase deficiency","category":"indication"},{"label":"Disorder of biliary tract","category":"indication"},{"label":"Mirum","category":"company"},{"label":"Approved 2010s","category":"decade"}],"phase":"marketed","safety":{"boxedWarnings":[],"safetySignals":[{"date":"","signal":"DIARRHOEA","source":"FDA FAERS","actionTaken":"56 reports"},{"date":"","signal":"DEATH","source":"FDA FAERS","actionTaken":"40 reports"},{"date":"","signal":"VOMITING","source":"FDA FAERS","actionTaken":"28 reports"},{"date":"","signal":"PYREXIA","source":"FDA FAERS","actionTaken":"20 reports"},{"date":"","signal":"SEIZURE","source":"FDA FAERS","actionTaken":"20 reports"},{"date":"","signal":"CONSTIPATION","source":"FDA FAERS","actionTaken":"19 reports"},{"date":"","signal":"ASPARTATE AMINOTRANSFERASE INCREASED","source":"FDA FAERS","actionTaken":"16 reports"},{"date":"","signal":"DISEASE PROGRESSION","source":"FDA FAERS","actionTaken":"16 reports"},{"date":"","signal":"ALANINE AMINOTRANSFERASE INCREASED","source":"FDA FAERS","actionTaken":"15 reports"},{"date":"","signal":"ABDOMINAL PAIN","source":"FDA FAERS","actionTaken":"13 reports"}],"drugInteractions":[{"drug":"cyclosporine","severity":"major","mechanism":"Inhibition of bile salt efflux pump (BSEP)","management":"Avoid concomitant use; if necessary, monitor serum transaminases and bilirubin","clinicalEffect":"Exacerbation of accumulation of conjugated bile salts in the liver, potentially leading to clinical symptoms"},{"drug":"bile acid binding resins (cholestyramine, colestipol, colesevelam)","severity":"moderate","mechanism":"Adsorption and reduction of bile acid absorption","management":"Take CHOLBAM at least 1 hour before or 4 to 6 hours (or at as great an interval as possible) after a bile acid binding resin","clinicalEffect":"Reduction in the efficacy of CHOLBAM"},{"drug":"aluminum-based antacids","severity":"moderate","mechanism":"Adsorption of bile acids in vitro","management":"Take CHOLBAM at least 1 hour before or 4 to 6 hours (or at as great an interval as possible) after an aluminum-based antacid","clinicalEffect":"Reduction in the availability of CHOLBAM"}],"commonSideEffects":[{"effect":"Diarrhea","drugRate":"2%","severity":"common","_validated":true},{"effect":"Reflux Esophagitis","drugRate":"1%","severity":"mild","_validated":true},{"effect":"Malaise","drugRate":"1%","severity":"mild","_validated":true},{"effect":"Jaundice","drugRate":"1%","severity":"mild","_validated":true},{"effect":"Skin lesion","drugRate":"1%","severity":"mild","_validated":true},{"effect":"Nausea","drugRate":"1%","severity":"mild","_validated":true},{"effect":"Abdominal Pain","drugRate":"1%","severity":"mild","_validated":true},{"effect":"Intestinal Polyp","drugRate":"1%","severity":"mild","_validated":true},{"effect":"Urinary Tract Infection","drugRate":"1%","severity":"mild","_validated":true},{"effect":"Peripheral Neuropathy","drugRate":"reported","severity":"unknown"}],"contraindications":["Appendicitis","Diarrhea","Disease of liver","Gallstone Complications","Gastrointestinal obstruction","Hypercholesterolemia","Incontinence of feces","Liver function tests abnormal","Obstruction of bile duct","Pregnancy, function","Primary biliary cirrhosis","Sclerosing cholangitis"],"specialPopulations":{"Pregnancy":"Pregnancy Exposure RegistryThere is pregnancy surveillance program that monitors pregnancy outcomes in women exposed to CHOLBAM during pregnancy [COCOA Registry (ChOlbam: Child and mOthers heAlth)]. Women who become pregnant during CHOLBAM treatment are encouraged to enroll. Patients or their health care provider should call 1-844-20C-OCOA or 1-844-202-6262 to enroll. Risk SummaryNo studies in pregnant women or animal reproduction studies have been conducted with CHOLBAM. Limited published case reports discuss pregnancies in women taking cholic acid for 3-HSD deficiency resulting in healthy infants. These reports may not adequately inform the presence or absence of drug-associated risk with the use of CHOLBAM during pregnancy. The background risk of major birth defects and miscarriage for the indicated population is unknown. However, the background risk in the U.S. general population of major birth defects is 2-4% and of miscarriage is 15-20% of clinically recognized pregnancies.","Geriatric use":"Clinical studies of CHOLBAM did not include any patients aged 65 years and over. It is not known if elderly patients respond differently from younger patients.","Paediatric use":"The safety and effectiveness of CHOLBAM have been established in pediatric patients weeks of age and older for the treatment of bile acid synthesis disorders due to SEDs and for adjunctive treatment of patients with PDs including Zellweger spectrum disorders who exhibit manifestations of liver disease, steatorrhea, or complications from decreased fat-soluble vitamin absorption [see Clinical Studies (14)]."}},"trials":[],"aliases":[],"company":"Mirum","patents":[],"pricing":[],"_sources":{"trials":{"url":"https://clinicaltrials.gov/search?intr=CHOLIC ACID","method":"api_direct","source":"ClinicalTrials.gov","rawText":"","confidence":1,"sourceType":"ctgov","retrievedAt":"2026-04-20T00:41:28.316075+00:00"},"regulatory.ca":{"url":"","method":"api_direct","source":"Health Canada DPD","rawText":"","confidence":1,"sourceType":"health_canada_dpd","retrievedAt":"2026-04-20T00:41:33.918358+00:00"},"regulatory.eu":{"url":"","method":"api_direct","source":"European Medicines Agency","rawText":"","confidence":1,"sourceType":"ema_api","retrievedAt":"2026-04-20T00:41:28.382923+00:00"},"regulatory.us":{"url":"","method":"api_direct","source":"FDA Drugs@FDA","rawText":"","confidence":1,"sourceType":"fda_drugsfda","retrievedAt":"2026-04-20T00:41:27.428971+00:00"},"publicationCount":{"url":"https://pubmed.ncbi.nlm.nih.gov/?term=CHOLIC ACID","method":"api_direct","source":"PubMed/NCBI","rawText":"","confidence":1,"sourceType":"pubmed","retrievedAt":"2026-04-20T00:41:34.271110+00:00"},"mechanism.drugClass":{"url":"https://api.fda.gov/drug/label.json","method":"deterministic","source":"FDA Label (EPC)","rawText":"","confidence":1,"sourceType":"fda_label","retrievedAt":"2026-04-20T00:41:26.927163+00:00"},"administration.route":{"url":"","method":"deterministic","source":"FDA Label","rawText":"","confidence":1,"sourceType":"fda_label","retrievedAt":"2026-04-20T00:41:26.927195+00:00"},"indications.approved":{"url":"","method":"ai_extraction","source":"FDA Label + AI","aiModel":"featherless","rawText":"","confidence":0.9,"sourceType":"fda_label","retrievedAt":"2026-04-20T00:42:11.633249+00:00"},"safety.boxedWarnings":{"url":"","method":"deterministic","source":"FDA Label (no boxed warning)","rawText":"","confidence":1,"sourceType":"fda_label","retrievedAt":"2026-04-20T00:41:26.927223+00:00"},"safety.safetySignals":{"url":"https://api.fda.gov/drug/event.json","method":"api_direct","source":"FDA FAERS","rawText":"","confidence":1,"sourceType":"fda_faers","retrievedAt":"2026-04-20T00:41:36.761746+00:00"},"safety.drugInteractions":{"url":"","method":"ai_extraction","source":"FDA Label + AI","aiModel":"featherless","rawText":"","confidence":0.9,"sourceType":"fda_label","retrievedAt":"2026-04-20T00:41:56.571596+00:00"},"crossReferences.chemblId":{"url":"https://www.ebi.ac.uk/chembl/compound_report_card/CHEMBL205596/","method":"api_direct","source":"ChEMBL (EMBL-EBI)","rawText":"","confidence":1,"sourceType":"chembl","retrievedAt":"2026-04-20T00:41:36.020523+00:00"},"regulatory.fda_application":{"url":"","method":"deterministic","source":"FDA Label","rawText":"NDA205750","confidence":1,"sourceType":"fda_label","retrievedAt":"2026-04-20T00:41:26.927231+00:00"}},"allNames":"cholbam","offLabel":[],"synonyms":["orphacol","cholic acid","cholalic acid","cholalin","colalin","kolbam","cholbam"],"timeline":[{"date":"2013-09-12","type":"positive","source":"DrugCentral","milestone":"EMA approval (Laboratoires CTRS)"},{"date":"2015-01-01","type":"neutral","source":"FDA Orange Book","milestone":"Rights transferred from RTRX to Mirum"},{"date":"2015-03-17","type":"positive","source":"DrugCentral","milestone":"FDA approval (Rtrx)"},{"date":"2023-03-27","type":"positive","source":"DrugCentral","milestone":"PMDA approval (ReqMed Company, Ltd)"}],"aiSummary":"Cholbam (Cholic Acid) is a bile acid medication originally developed by RTRX and currently owned by Mirum. It targets the G-protein coupled bile acid receptor 1 and is classified as a bile acid modality. Cholbam is FDA-approved to treat various conditions, including inborn errors of bile acid metabolism, disorders of the biliary tract, and incomplete passage of stool. The medication is off-patent, with no active Orange Book patents. As a bile acid, Cholbam works by regulating the levels of bile acids in the body.","approvals":[{"date":"2013-09-12","orphan":true,"company":"Laboratoires CTRS","regulator":"EMA"},{"date":"2015-03-17","orphan":true,"company":"RTRX","regulator":"FDA"},{"date":"2023-03-27","orphan":true,"company":"ReqMed Company, Ltd","regulator":"PMDA"}],"brandName":"Cholbam","ecosystem":[{"indication":"3-Beta-hydroxy-delta-5-C27-steroid dehydrogenase deficiency","otherDrugs":[],"globalPrevalence":null},{"indication":"Calculus in biliary tract","otherDrugs":[{"name":"chenodiol","slug":"chenodiol","company":""},{"name":"ursodiol","slug":"ursodiol","company":"Actavis Labs Ut Inc"}],"globalPrevalence":null},{"indication":"Cholestanol storage disease","otherDrugs":[],"globalPrevalence":null},{"indication":"Constipation","otherDrugs":[{"name":"bisacodyl","slug":"bisacodyl","company":"Braintree"},{"name":"docusate sodium","slug":"docusate-sodium","company":""},{"name":"glycerol","slug":"glycerol","company":"B Braun"},{"name":"homatropine","slug":"homatropine","company":""}],"globalPrevalence":null},{"indication":"Delta-4-3-oxosteroid-5-beta-reductase deficiency","otherDrugs":[],"globalPrevalence":null},{"indication":"Disorder of biliary tract","otherDrugs":[{"name":"homatropine","slug":"homatropine","company":""}],"globalPrevalence":null},{"indication":"Inborn error of bile acid metabolism","otherDrugs":[],"globalPrevalence":null},{"indication":"Incomplete passage of stool","otherDrugs":[{"name":"bisacodyl","slug":"bisacodyl","company":"Braintree"},{"name":"homatropine","slug":"homatropine","company":""},{"name":"macrogol","slug":"macrogol","company":"Braintree"},{"name":"magnesium carbonate","slug":"magnesium-carbonate","company":"United Guardian"}],"globalPrevalence":null}],"mechanism":{"target":"G-protein coupled bile acid receptor 1","novelty":"Follow-on","targets":[{"gene":"GPBAR1","source":"DrugCentral","target":"G-protein coupled bile acid receptor 1","protein":"G-protein coupled bile acid receptor 1"},{"gene":"NR1H4","source":"DrugCentral","target":"Bile acid receptor","protein":"Bile acid receptor"}],"modality":"Small Molecule","drugClass":"Bile Acid [EPC]","explanation":"Cholic acid is primary bileacid synthesized from cholesterol in the liver. In bile acid synthesisdisorders due to SEDs in the biosynthetic pathway, and in PDs includingZellweger spectrum disorders, deficiency of primary bile acids leadsto unregulated accumulation of intermediate bile acids and cholestasis. Bile acids facilitate fat digestion and absorption by forming mixedmicelles and facilitate absorption of fat-soluble vitamins in theintestine.Endogenousbile acids including cholic acid enhance bile flow and provide thephysiologic feedback inhibition of bile acid synthesis. The mechanismof action of cholic acid has not been fully established; however,it is known that cholic acid and its conjugates are endogenous ligandsof the nuclear receptor, farnesoid receptor (FXR). FXR regulatesenzymes and transporters that are involved in bile acid synthesisand in the enterohepatic circulation to maintain bile acid homeostasisunder normal physiologic conditions.","oneSentence":"Cholbam works by binding to the G-protein coupled bile acid receptor 1, which helps regulate bile acid levels in the body.","technicalDetail":"Cholbam, a small molecule bile acid, acts as a positive allosteric modulator of the G-protein coupled bile acid receptor 1 (TGR5), which plays a crucial role in regulating bile acid levels and promoting bile acid synthesis."},"commercial":{"launchDate":"2015","_launchSource":"DrugCentral (FDA 2015-03-17, RTRX)"},"references":[{"id":1,"url":"https://drugcentral.org/drugcard/3096","fields":["approvals","synonyms","ATC","PK","indications","contraindications","DDIs","targets","patents","FAERS"],"source":"DrugCentral"},{"id":2,"url":"https://clinicaltrials.gov/search?intr=CHOLIC%20ACID","fields":["trials"],"source":"ClinicalTrials.gov"},{"id":3,"url":"https://pubmed.ncbi.nlm.nih.gov/?term=CHOLIC ACID","fields":["publications"],"source":"PubMed/NCBI"},{"id":4,"url":"https://www.fda.gov/drugs/drug-approvals-and-databases/orange-book-data-files","fields":["patents","exclusivity","genericManufacturers"],"source":"FDA Orange Book"}],"_emaChecked":true,"_enrichedAt":"2026-03-30T09:36:48.874874","_validation":{"fieldsValidated":1,"lastValidatedAt":"2026-04-20T00:42:11.779630+00:00","fieldsConflicting":0,"overallConfidence":0.95},"biosimilars":[],"competitors":[{"drugName":"chenodiol","drugSlug":"chenodiol","fdaApproval":"1983-07-28","genericCount":1,"patentStatus":"Off-patent — generic available","relationship":"same-class"},{"drugName":"ursodiol","drugSlug":"ursodiol","fdaApproval":"1987-12-31","genericCount":19,"patentStatus":"Off-patent — generic available","relationship":"same-class"},{"drugName":"obeticholic acid","drugSlug":"obeticholic-acid","fdaApproval":"2016-05-27","patentExpiry":"Sep 6, 2033","patentStatus":"Patent protected","relationship":"same-class"}],"genericName":"cholic acid","indications":{"approved":[{"id":"cholic-acid-bile-acid-synthesis-disorders-","name":"Bile Acid Synthesis Disorders Due to SEDs","dosing":null,"approvals":[],"diseaseId":"","eligibility":"Patients with bile acid synthesis disorders due to single enzyme defects","pivotalTrial":null,"restrictions":[],"patientPopulation":"Patients with bile acid synthesis disorders due to single enzyme defects","diagnosticRequired":null,"brandNameForIndication":"Cholbam"},{"id":"cholic-acid-adjunctive-treatment-of-peroxi","name":"Adjunctive Treatment of Peroxisomal Disorders","dosing":null,"approvals":[],"diseaseId":"","eligibility":"Patients with peroxisomal disorders including Zellweger spectrum disorders exhibiting liver disease, steatorrhea, or complications from decreased fat-soluble vitamin absorption","pivotalTrial":null,"restrictions":[],"patientPopulation":"Patients with peroxisomal disorders including Zellweger spectrum disorders exhibiting liver disease, steatorrhea, or complications from decreased fat-soluble vitamin absorption","diagnosticRequired":null,"brandNameForIndication":"Cholbam"}],"offLabel":[],"pipeline":[]},"currentOwner":"Mirum","drugCategory":"active","labelChanges":[],"patentStatus":"Off-patent — no active Orange Book patents","relatedDrugs":[{"drugId":"chenodiol","brandName":"chenodiol","genericName":"chenodiol","approvalYear":"1983","relationship":"same-class"},{"drugId":"ursodiol","brandName":"ursodiol","genericName":"ursodiol","approvalYear":"1987","relationship":"same-class"},{"drugId":"obeticholic-acid","brandName":"obeticholic acid","genericName":"obeticholic acid","approvalYear":"2016","relationship":"same-class"}],"trialDetails":[{"nctId":"NCT06783621","phase":"PHASE4","title":"A Study to Evaluate Patient Satisfaction With the Overall Face and Neck Appearance After Combined Treatment of OnabotulinumtoxinA, JUVÉDERM® Products, KYBELLA, CoolSculpting Elite and SkinMedica Products","status":"COMPLETED","sponsor":"AbbVie","startDate":"2025-01-16","conditions":["Facial Contouring"],"enrollment":130,"completionDate":"2026-02-26"},{"nctId":"NCT02400216","phase":"","title":"Deciphering the Mechanisms Involved in Microbial Translocation Across the Spectrum of HCV Associated Liver Fibrosis","status":"COMPLETED","sponsor":"National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)","startDate":"2015-05-29","conditions":["Cirrhosis"],"enrollment":30,"completionDate":"2017-04-25"},{"nctId":"NCT06517862","phase":"PHASE4","title":"Efficacy & Safety of Oral Adjuvants to Phototherapy in Neonatal Hyperbilirubinemia","status":"RECRUITING","sponsor":"Amira Adel Fouly","startDate":"2024-09-01","conditions":["Neonatal Hyperbilirubinemia"],"enrollment":80,"completionDate":"2026-06-30"},{"nctId":"NCT07473817","phase":"","title":"Validation of Different Methods for HepQuant DuO® Blood Sample Collection","status":"NOT_YET_RECRUITING","sponsor":"HepQuant, LLC","startDate":"2026-04-20","conditions":["Healthy Adult Participants","Liver Disease Chronic"],"enrollment":200,"completionDate":"2027-04-20"},{"nctId":"NCT03724175","phase":"PHASE2,PHASE3","title":"The Role of Secondary Bile Acids in Intestinal Inflammation","status":"TERMINATED","sponsor":"Stanford University","startDate":"2019-08-26","conditions":["Ulcerative Colitis","Pouchitis"],"enrollment":2,"completionDate":"2026-03-02"},{"nctId":"NCT01907074","phase":"PHASE2","title":"Quantitative Liver Function Tests Using Cholates","status":"TERMINATED","sponsor":"Baylor Research Institute","startDate":"2011-06-23","conditions":["End Stage Liver Disease"],"enrollment":103,"completionDate":"2025-05-20"},{"nctId":"NCT07378761","phase":"PHASE2","title":"Comparing UDCA and Corticosteroids in Immunotherapy Induced Cholestatic Hepatitis","status":"NOT_YET_RECRUITING","sponsor":"University Hospital, Montpellier","startDate":"2026-02","conditions":["Immune-Mediated Cholestasis"],"enrollment":94,"completionDate":"2028-08"},{"nctId":"NCT05751967","phase":"PHASE3","title":"Fenofibrate Combined With Ursodeoxycholic Acid in Subjects With Primary Biliary Cholangitis","status":"RECRUITING","sponsor":"Xijing Hospital of Digestive Diseases","startDate":"2023-02-22","conditions":["Primary Biliary Cholangitis"],"enrollment":150,"completionDate":"2027-12-01"},{"nctId":"NCT06755541","phase":"PHASE3","title":"Fenofibrate in Combination With Ursodeoxycholic Acid in Primary Biliary Cholangitis: a Real World Study","status":"RECRUITING","sponsor":"Xijing Hospital of Digestive Diseases","startDate":"2024-01-30","conditions":["Primary Biliary Cholangitis (PBC)"],"enrollment":150,"completionDate":"2035-12"},{"nctId":"NCT00200343","phase":"PHASE3","title":"Efficacy and Safety Study of Ursodeoxycholic Acid to Treat Chronic Hepatitis C","status":"COMPLETED","sponsor":"Tanabe Pharma Corporation","startDate":"2002-07","conditions":["Chronic Hepatitis C"],"enrollment":596,"completionDate":"2004-12"},{"nctId":"NCT07296458","phase":"PHASE3","title":"FIREFLY Trial: Fenofibrate Intervention---Randomized Evaluation in First-Line PBC Therapy","status":"RECRUITING","sponsor":"Xijing Hospital of Digestive Diseases","startDate":"2025-12-15","conditions":["Primary Biliary Cholangitis (PBC)"],"enrollment":132,"completionDate":"2028-12-31"},{"nctId":"NCT06091787","phase":"NA","title":"Effect of Ursodeoxycholic Acid Supplementation on Liver Regeneration on Right Lobe Donor Hepatectomy","status":"COMPLETED","sponsor":"Institute of Liver and Biliary Sciences, India","startDate":"2023-09-01","conditions":["Liver Regeneration","Ursodeoxycholic Acid","Partial Liver Resection"],"enrollment":90,"completionDate":"2024-11-30"},{"nctId":"NCT06922669","phase":"NA","title":"Glucocorticoids for Acute Drug Induced Liver Injury With Hyperbilirubinemia","status":"RECRUITING","sponsor":"General Hospital of Shenyang Military Region","startDate":"2025-06-24","conditions":["Drug Induced Liver Injury"],"enrollment":232,"completionDate":"2027-09-30"},{"nctId":"NCT07273734","phase":"NA","title":"UDCA to Prevent Post-TIPS Hepatic Encephalopathy","status":"NOT_YET_RECRUITING","sponsor":"West China Hospital","startDate":"2026-01-01","conditions":["Hepatic Encephalopathy (HE)","TIPS"],"enrollment":270,"completionDate":"2027-12-31"},{"nctId":"NCT07110987","phase":"NA","title":"The Efficacy of Ursodeoxycholic Acid (UDCA) as Adjuvant Therapy to Phototherapy in the Management of Neonatal Indirect Hyperbilirubinemia","status":"RECRUITING","sponsor":"Tishreen University Hospital","startDate":"2025-06-26","conditions":["Hyperbilirubinemia, Neonatal Indirect"],"enrollment":70,"completionDate":"2027-03-01"},{"nctId":"NCT06610760","phase":"PHASE4","title":"Efficacy and Safety of Ursodeoxycholic Acid in Reversing Gastric Intestinal Metaplasia","status":"RECRUITING","sponsor":"Yongquan Shi","startDate":"2024-11-12","conditions":["Gastric Intestinal Metaplasia"],"enrollment":196,"completionDate":"2026-10-31"},{"nctId":"NCT07237425","phase":"PHASE1","title":"Intralesional Ronkyla Plus Injection for the Treatment of Superficial Lipoma","status":"RECRUITING","sponsor":"Glonova Pharma Co., Ltd","startDate":"2025-11-14","conditions":["Lipoma","Submental Fullness"],"enrollment":56,"completionDate":"2027-01-14"},{"nctId":"NCT06591468","phase":"PHASE2,PHASE3","title":"A Multicenter, Randomized, Controlled Trial of Prednisone Combined With Ursodeoxycholic Acid in the Treatment of Primary Biliary Cholangitis With Moderate to Severe Interface Hepatitis Characteristics","status":"ENROLLING_BY_INVITATION","sponsor":"Han Ying","startDate":"2024-10-30","conditions":["Primary Biliary Cholangitis (PBC)"],"enrollment":244,"completionDate":"2027-12-31"},{"nctId":"NCT06591455","phase":"EARLY_PHASE1","title":"A Pilot Study of Fenofibrate and Ursodeoxycholic Acid in the Treatment of Newly Diagnosed Primary Biliary Cholangitis","status":"COMPLETED","sponsor":"Han Ying","startDate":"2024-09-14","conditions":["Primary Biliary Cholangitis (PBC)"],"enrollment":30,"completionDate":"2025-08-18"},{"nctId":"NCT06132165","phase":"PHASE1","title":"Efficacy of Skin Cooling in Reducing Pain Associated With Non-invasive Treatments of Neurofibromatosis Type 1 Cutaneous Neurofibromas","status":"COMPLETED","sponsor":"Massachusetts General Hospital","startDate":"2024-03-01","conditions":["Neurofibromatosis 1"],"enrollment":13,"completionDate":"2025-07-31"},{"nctId":"NCT06300502","phase":"PHASE1","title":"Assessing the Efficacy of Repeat, Monthly Treatments of Deoxycholate for NF1 Associated Cutaneous Neurofibromas (cNFs)","status":"ENROLLING_BY_INVITATION","sponsor":"Massachusetts General Hospital","startDate":"2025-04-08","conditions":["Neurofibromas, Cutaneous","Neurofibromatosis 1"],"enrollment":15,"completionDate":"2026-02"},{"nctId":"NCT06884748","phase":"EARLY_PHASE1","title":"Prevention of Recurrence of Clostridioides Difficile Colitis With Ursodeoxycholic Acid (UCDA) as a Supplement to Standard Therapy","status":"NOT_YET_RECRUITING","sponsor":"Medical College of Wisconsin","startDate":"2025-12-01","conditions":["Clostridioides Difficile Infection"],"enrollment":30,"completionDate":"2028-05-01"},{"nctId":"NCT06684106","phase":"NA","title":"Ursodeoxycholic Acid Attenuates Statin-Induced Impaired Glucose Tolerance","status":"RECRUITING","sponsor":"First Affiliated Hospital Xi'an Jiaotong University","startDate":"2024-11-25","conditions":["Hyperlipidemia","Ursodeoxycholic Acid","Statin Therapy","Glucose Intolerance"],"enrollment":128,"completionDate":"2027-06-01"},{"nctId":"NCT07009132","phase":"","title":"Cholate Clearance in Fontan and Heart Failure","status":"RECRUITING","sponsor":"HepQuant, LLC","startDate":"2025-08-04","conditions":["Fontan","Right Heart Failure"],"enrollment":75,"completionDate":"2031-12"},{"nctId":"NCT02314208","phase":"PHASE2","title":"Therapeutic Metabolic Intervention in Patients With Spastic Paraplegia SPG5","status":"COMPLETED","sponsor":"Institut National de la Santé Et de la Recherche Médicale, France","startDate":"2015-01-08","conditions":["Spastic Paraplegia, Hereditary"],"enrollment":12,"completionDate":"2018-01-01"},{"nctId":"NCT07102979","phase":"NA","title":"Remedial Mechanism of Simvastatin and Ursodeoxycholic Acid in Liver Cirrhosis: Crosstalk of Bile Secretion, Gut Microbiome, and Host Immune Response","status":"ENROLLING_BY_INVITATION","sponsor":"Chang Gung Memorial Hospital","startDate":"2025-09-01","conditions":["Liver Cirrhosis"],"enrollment":150,"completionDate":"2025-12-31"},{"nctId":"NCT00457639","phase":"PHASE2","title":"Cholic Acid for Hepatic Steatosis in Lipodystrophy","status":"COMPLETED","sponsor":"University of Texas Southwestern Medical Center","startDate":"2006-04","conditions":["Hepatic Steatosis"],"enrollment":18,"completionDate":"2011-04"},{"nctId":"NCT07068191","phase":"NA","title":"Gepaktiv vs UDCA and Ademetionine in MAFLD With Hepatomegaly","status":"RECRUITING","sponsor":"Phenomen Pharma","startDate":"2025-06-19","conditions":["Metabolic Dysfunction-associated Fatty Liver Disease (MAFLD)","Hepatomegaly","Nonalcoholic Fatty Liver (NAFL)","Nonalcoholic Fatty Liver Disease (NAFLD)","Fatty Liver","Fatty Liver, Alcoholic","Fatty Liver, Nonalcoholic","Fatty Liver Disease"],"enrollment":90,"completionDate":"2025-08"},{"nctId":"NCT06899152","phase":"NA","title":"HepQuant: Study to Assess the Role of Blood-based Biomarkers and Quantitative MR Imaging for Patients Receiving Radiation Therapy for Liver Cancer","status":"RECRUITING","sponsor":"Montefiore Medical Center","startDate":"2025-07-16","conditions":["Liver Cancer","Hepatocellular Carcinoma","Hepatocellular Cancer","Cholangiocarcinoma","Liver Metastases"],"enrollment":40,"completionDate":"2031-07"},{"nctId":"NCT07072949","phase":"PHASE1","title":"The Impact of Ursodeoxycholic Acid and Probiotics on Metabolic Outcomes in Type 2 Diabetic Patients Taking Metformin","status":"COMPLETED","sponsor":"University of Banja Luka","startDate":"2023-01-03","conditions":["Type 2 Diabetes Mellitus"],"enrollment":90,"completionDate":"2024-01-31"},{"nctId":"NCT00873275","phase":"PHASE1","title":"Ursodiol, Combination Chemotherapy, and Bevacizumab in Treating Patients With Stage IV Colorectal Cancer","status":"COMPLETED","sponsor":"City of Hope Medical Center","startDate":"2009-03-11","conditions":["Colorectal Cancer"],"enrollment":11,"completionDate":"2025-02-26"},{"nctId":"NCT06294847","phase":"PHASE3","title":"Ursodeoxycholic Acid (UDCA) as a Neuroprotective Adjuvant Treatment to Rhegmatogenous Retinal Detachment Surgery","status":"RECRUITING","sponsor":"Hopital Foch","startDate":"2024-08-20","conditions":["Retinal Detachment"],"enrollment":120,"completionDate":"2027-11"},{"nctId":"NCT03284034","phase":"NA","title":"Cryolipolysis Versus ATX-101 (Deoxycholic Acid) for Upper Back Fat","status":"TERMINATED","sponsor":"Northwestern University","startDate":"2017-11-27","conditions":["Upper Back Fat"],"enrollment":8,"completionDate":"2020-06-08"},{"nctId":"NCT04407650","phase":"PHASE4","title":"Ursodeoxycholic Acid vs Metformin in Gestational Diabetes Mellitus","status":"ACTIVE_NOT_RECRUITING","sponsor":"King's College London","startDate":"2021-07-01","conditions":["Gestational Diabetes"],"enrollment":64,"completionDate":"2025-05-31"},{"nctId":"NCT03583996","phase":"NA","title":"The SHUNT-V Study for Varices","status":"COMPLETED","sponsor":"HepQuant, LLC","startDate":"2019-01-23","conditions":["Chronic Liver Disease"],"enrollment":306,"completionDate":"2022-10-22"},{"nctId":"NCT06918080","phase":"PHASE4","title":"Hepatoprotective Measures for Children at High Risk of NAFLD","status":"ACTIVE_NOT_RECRUITING","sponsor":"Ain Shams University","startDate":"2023-11-19","conditions":["Non Alcoholic Fatty Liver Diseases (NAFLD)","Obesity and Overweight","Acute Lymphoblastic Leukemia"],"enrollment":200,"completionDate":"2025-07"},{"nctId":"NCT04650243","phase":"PHASE4","title":"Clinical Research of Tapering UDCA in PBC Patients With a Complete Response","status":"RECRUITING","sponsor":"Peking Union Medical College Hospital","startDate":"2020-01-21","conditions":["Primary Biliary Cholangitis"],"enrollment":90,"completionDate":"2025-05"},{"nctId":"NCT03188146","phase":"","title":"Performance of Scoring Systems in Chinese Patients With Primary Biliary Cholangitis (PBC) on Ursodeoxycholic Acid","status":"COMPLETED","sponsor":"Humanity and Health Research Centre","startDate":"2017-05-01","conditions":["Primary Biliary Cholangitis (PBC)"],"enrollment":57,"completionDate":"2025-02-15"},{"nctId":"NCT04924868","phase":"PHASE3","title":"Ursodeoxycolic Acid for the Prevention of Relapsing Complications After Gallstone Acute Pancreatitis","status":"RECRUITING","sponsor":"Hospital General Universitario de Alicante","startDate":"2021-11-10","conditions":["Acute Pancreatitis Due to Gallstones"],"enrollment":332,"completionDate":"2027-07-01"},{"nctId":"NCT06842472","phase":"PHASE2","title":"PD-L1 Inhibitor + RT ± Ursodeoxycholic Acid in Recurrent/Metastatic HER2-Neg Breast Cancer","status":"NOT_YET_RECRUITING","sponsor":"Cancer Institute and Hospital, Chinese Academy of Medical Sciences","startDate":"2025-02","conditions":["Breast Cancer"],"enrollment":70,"completionDate":"2029-12"},{"nctId":"NCT05659654","phase":"PHASE1,PHASE2","title":"The Application of Ursodeoxycholic Acid for the Prevention of SARS-CoV-2 Infection (COVID-19)","status":"COMPLETED","sponsor":"Institute of Hematology & Blood Diseases Hospital, China","startDate":"2022-12-16","conditions":["COVID-19"],"enrollment":95,"completionDate":"2023-03-08"},{"nctId":"NCT04730583","phase":"PHASE1","title":"Tolerability of Device Based Therapies for Neurofibromatosis Type 1 Cutaneous Neurofibromas","status":"COMPLETED","sponsor":"Massachusetts General Hospital","startDate":"2021-06-02","conditions":["Cutaneous Neurofibroma"],"enrollment":34,"completionDate":"2024-04-19"},{"nctId":"NCT06120036","phase":"PHASE1","title":"Dosing and Tolerability of Deoxycholic Acid vs. Polidocanol in the Treatment of Neurofibromatosis Type 1 Cutaneous Neurofibromas","status":"COMPLETED","sponsor":"Massachusetts General Hospital","startDate":"2022-12-06","conditions":["Neurofibromatosis 1"],"enrollment":20,"completionDate":"2025-01-09"},{"nctId":"NCT06639425","phase":"NA","title":"Efficacy of Lactobacillus Paracasei LC19 on Type 2 Diabetes","status":"RECRUITING","sponsor":"Beijing Chao Yang Hospital","startDate":"2024-10-12","conditions":["Type 2 Diabetes"],"enrollment":60,"completionDate":"2026-12-31"},{"nctId":"NCT06260748","phase":"PHASE3","title":"A Study of Chenodeoxycholic Acid (CDCA) in Newly Diagnosed Participants With Cerebrotendinous Xanthomatosis (CTX)","status":"WITHDRAWN","sponsor":"Leadiant Biosciences, Inc.","startDate":"2024-05-27","conditions":["Cerebrotendinous Xanthomatoses"],"enrollment":0,"completionDate":"2025-12-31"},{"nctId":"NCT05925309","phase":"NA","title":"Preventive Effect of Prophylactic Oral Antibiotics Against Cholangitis After Kasai Portoenterostomy","status":"RECRUITING","sponsor":"Children's Hospital of Fudan University","startDate":"2023-07-01","conditions":["Biliary Atresia","Cholangitis","Anti-Bacterial Agents"],"enrollment":356,"completionDate":"2027-07-31"},{"nctId":"NCT06629909","phase":"PHASE1","title":"Safety and Feasibility of Human Umbilical Cord Mesenchymal Stem Cell-Derived Secretome in the Treatment of Liver Cirrhosis: a Comprehensive Evaluation of Fibrosis Reduction, Immunomodulation, and Hepatic Regeneration: a Single Center, Randomized, Phase I Clinical Trial","status":"NOT_YET_RECRUITING","sponsor":"Baermed","startDate":"2024-12-01","conditions":["Liver Cirrhosis"],"enrollment":54,"completionDate":"2026-10-31"},{"nctId":"NCT00909610","phase":"PHASE1","title":"Ursodiol Tablets 500 mg Under Fasting Conditions","status":"COMPLETED","sponsor":"Teva Pharmaceuticals USA","startDate":"2006-12","conditions":["Healthy"],"enrollment":80,"completionDate":"2007-01"},{"nctId":"NCT00909753","phase":"PHASE1","title":"Ursodiol 500 mg Tablets Under Fed Conditions","status":"COMPLETED","sponsor":"Teva Pharmaceuticals USA","startDate":"2006-09","conditions":["Healthy"],"enrollment":92,"completionDate":"2006-10"},{"nctId":"NCT04640571","phase":"PHASE4","title":"Impact of Metformin and Polysorbate 80 on Drug Absorption and Disposition","status":"COMPLETED","sponsor":"University of Maryland, Baltimore","startDate":"2021-04-01","conditions":["Bile Salt Export Pump (BSEP) Transporter","Polysorbate 80"],"enrollment":18,"completionDate":"2021-11-01"},{"nctId":"NCT03004118","phase":"PHASE4","title":"Therapeutic Effect of Ursodeoxycholic Acid in Functional Dyspepsia","status":"ACTIVE_NOT_RECRUITING","sponsor":"Universitaire Ziekenhuizen KU Leuven","startDate":"2017-01","conditions":["Functional Dyspepsia"],"enrollment":16,"completionDate":"2025-11"},{"nctId":"NCT06278090","phase":"","title":"Impact of Ursodeoxycholic Acid Treatment on the Gallbladder Polyp Evolution","status":"RECRUITING","sponsor":"Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz","startDate":"2024-03-01","conditions":["Gallbladder Polyp"],"enrollment":36,"completionDate":"2026-07"},{"nctId":"NCT05973370","phase":"PHASE2","title":"Evaluating the Effect of Ursodeoxycholic Acid in Patients With Rheumatoid Arthritis","status":"COMPLETED","sponsor":"Tanta University","startDate":"2023-05-01","conditions":["Rheumatoid Arthritis"],"enrollment":60,"completionDate":"2024-05-30"},{"nctId":"NCT03720990","phase":"PHASE1,PHASE2","title":"Smith-Lemli-Opitz Syndrome and Cholic Acid","status":"COMPLETED","sponsor":"University of Nebraska","startDate":"2021-03-27","conditions":["Smith-Lemli-Opitz Syndrome"],"enrollment":12,"completionDate":"2023-09-30"},{"nctId":"NCT05526807","phase":"NA","title":"Ursodeoxycholic Acid in C. Difficile Infection","status":"TERMINATED","sponsor":"Nottingham University Hospitals NHS Trust","startDate":"2019-05-08","conditions":["Clostridioides Difficile Infection"],"enrollment":6,"completionDate":"2023-12-31"},{"nctId":"NCT01505569","phase":"NA","title":"Auto Transplant for High Risk or Relapsed Solid or CNS Tumors","status":"COMPLETED","sponsor":"Masonic Cancer Center, University of Minnesota","startDate":"2011-10-20","conditions":["Ewing's Family Tumors","Renal Tumors","Hepatoblastoma","Rhabdomyosarcoma","Soft Tissue Sarcoma","Primary Malignant Brain Neoplasms","Retinoblastoma","Medulloblastoma","Supra-tentorial Primative Neuro-Ectodermal Tumor (PNET)","Atypical Teratoid/Rhabdoid Tumor (AT/RT)","CNS Tumors","Germ Cell Tumors"],"enrollment":44,"completionDate":"2024-02-01"},{"nctId":"NCT00550862","phase":"PHASE2","title":"Study of INT 747 in Combination With URSO in Patients With Primay Biliary Cirrhosis (PBC)","status":"TERMINATED","sponsor":"Intercept Pharmaceuticals","startDate":"2007-10","conditions":["Liver Cirrhosis, Biliary"],"enrollment":165,"completionDate":"2010-12"},{"nctId":"NCT06180057","phase":"PHASE1","title":"Bioequivalence Study to Compare Chenodeoxycholic Acid Capsules (250mg Chenodeoxycholic Acid) Versus Chenodeoxycholic Acid Leadiant 250 mg Hard Capsules (250mg Chenodeoxycholic Acid)","status":"COMPLETED","sponsor":"Humanis Saglık Anonim Sirketi","startDate":"2022-06-12","conditions":["Cerebrotendinous Xanthomatoses"],"enrollment":24,"completionDate":"2022-08-15"},{"nctId":"NCT06127355","phase":"PHASE3","title":"Gestational Treatment With Ursodeoxycholic Acid Compared to Placebo to Reduce Severity of Gestational Diabetes Mellitus Diagnosed at 24-28 Weeks' Gestation","status":"COMPLETED","sponsor":"Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia","startDate":"2021-03-03","conditions":["Gestational Diabetes"],"enrollment":113,"completionDate":"2022-05-10"},{"nctId":"NCT01115582","phase":"PHASE3","title":"Efficacy of To Be Marketed (TBM) Cholic Acid Capsules Used to Treat Children With Inborn Errors of Bile Acid Synthesis","status":"COMPLETED","sponsor":"Mirum Pharmaceuticals, Inc.","startDate":"2010-04","conditions":["Inborn Errors of Bile Acid Synthesis"],"enrollment":16,"completionDate":"2010-08"},{"nctId":"NCT01438411","phase":"PHASE3","title":"Open Label, Continuation Study of Cholic Acid in Subjects With Inborn Errors of Bile Acid Synthesis","status":"COMPLETED","sponsor":"Mirum Pharmaceuticals, Inc.","startDate":"2010-01","conditions":["Bile Acid Synthesis Defect"],"enrollment":53,"completionDate":"2016-07"},{"nctId":"NCT00007020","phase":"PHASE3","title":"Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid","status":"COMPLETED","sponsor":"Mirum Pharmaceuticals, Inc.","startDate":"1992-01","conditions":["Infantile Refsum's Disease","Zellweger Syndrome","Adrenoleukodystrophy","Peroxisomal Disorders","Cholestasis"],"enrollment":85,"completionDate":"2009-12"},{"nctId":"NCT03115086","phase":"","title":"The REPLACE Registry for Cholbam® (Cholic Acid)","status":"ACTIVE_NOT_RECRUITING","sponsor":"Mirum Pharmaceuticals, Inc.","startDate":"2017-07-10","conditions":["Bile Acid Synthesis Disorders"],"enrollment":55,"completionDate":"2039-07"},{"nctId":"NCT05902468","phase":"PHASE2,PHASE3","title":"Ursodeoxycholic Acid as add-on Therapy in Type 2 Diabetes Mellitus","status":"UNKNOWN","sponsor":"Tanta University","startDate":"2023-06-28","conditions":["Diabetes Mellitus, Type 2"],"enrollment":88,"completionDate":"2024-11-28"},{"nctId":"NCT03602976","phase":"PHASE2","title":"Ursodeoxycholic Acid (UDCA) for Hepatic Sarcoidosis","status":"TERMINATED","sponsor":"Ethan Weinberg","startDate":"2018-08-20","conditions":["Hepatic Sarcoidosis, Elevated Alkaline Phosphatase"],"enrollment":7,"completionDate":"2022-01-12"},{"nctId":"NCT04076527","phase":"","title":"Prospective, Multicenter Cohort Study on Primary Biliary Cholangitis","status":"UNKNOWN","sponsor":"University of Leipzig","startDate":"2019-09-19","conditions":["PBC","Primary Biliary Cholangitis"],"enrollment":1200,"completionDate":"2024-03"},{"nctId":"NCT05874336","phase":"PHASE1","title":"An Open-Label, Two-Part Study Designed to Assess the Absolute Bioavailability and Mass Balance of Aramchol","status":"COMPLETED","sponsor":"Galmed Pharmaceuticals Ltd","startDate":"2020-06-17","conditions":["NASH"],"enrollment":6,"completionDate":"2020-08-11"},{"nctId":"NCT05416580","phase":"PHASE3","title":"Efficacy of Ursodeoxycholic Acid (UDCA) in Patients With Type 2 Diabetes","status":"UNKNOWN","sponsor":"University of Banja Luka","startDate":"2022-09-12","conditions":["Diabetes Mellitus, Type 2"],"enrollment":60,"completionDate":"2024-04"},{"nctId":"NCT05849558","phase":"PHASE4","title":"Study to Compare the Efficacy & Safety of Ursoplus Capsules vs. UDCA vs. Placebo Among Chronic Liver Disease Patients","status":"UNKNOWN","sponsor":"MinaPharm Pharmaceuticals","startDate":"2022-02-22","conditions":["Chronic Liver Disease"],"enrollment":297,"completionDate":"2023-12-31"},{"nctId":"NCT05832697","phase":"PHASE1","title":"An Exploratory Clinical Trial to Evaluate and Compare Safety and Pharmacokinetic Characteristics After Administration of the DWJ1439, DWJ1464, DWC202108 or DWC202109 in Healthy Adult Volunteers","status":"COMPLETED","sponsor":"Daewoong Pharmaceutical Co. LTD.","startDate":"2022-01-27","conditions":["Healthy Subject"],"enrollment":44,"completionDate":"2023-01-30"},{"nctId":"NCT05812612","phase":"","title":"UDCA in the Treatment of COVID-19 Infection and Its Clinical Prognosis in Patients With Autoimmune Liver Disease","status":"UNKNOWN","sponsor":"Beijing Ditan Hospital","startDate":"2023-01-30","conditions":["COVID-19 Infection"],"enrollment":2000,"completionDate":"2023-12-30"},{"nctId":"NCT05690646","phase":"PHASE4","title":"Prophylactic Effect of Nirmatrelvir/Ritonavir and Ursodeoxycholic Acid on Reducing Complications After Cardiac Surgery","status":"UNKNOWN","sponsor":"China National Center for Cardiovascular Diseases","startDate":"2023-01-28","conditions":["Cardiac Surgery"],"enrollment":491,"completionDate":"2026-01-06"},{"nctId":"NCT04834557","phase":"PHASE2","title":"Evaluating the Effect of Digoxin and Ursodeoxycholic Acid in Patients With Rheumatoid Arthritis","status":"COMPLETED","sponsor":"Tanta University","startDate":"2021-11-01","conditions":["Rheumatoid Arthritis"],"enrollment":90,"completionDate":"2022-09-30"},{"nctId":"NCT04542473","phase":"PHASE2,PHASE3","title":"Pancreatic Enzymes and Bile Acids in Acutely Ill Severely Malnourished Children","status":"UNKNOWN","sponsor":"University of Oxford","startDate":"2021-07-01","conditions":["SEPSIS","MALNUTRITION, CHILD"],"enrollment":400,"completionDate":"2024-06-30"},{"nctId":"NCT04531878","phase":"PHASE2,PHASE3","title":"BSEP Function Rescue During Childhood Inhereditary Cholestatic Diseases","status":"WITHDRAWN","sponsor":"Children's Hospital of Fudan University","startDate":"2023-02-08","conditions":["Hereditary Diseases","Cholestasis, Intrahepatic"],"enrollment":0,"completionDate":"2023-02-08"},{"nctId":"NCT05467553","phase":"PHASE2","title":"A Study to Compare P1101 Plus TAF With or Without UDCA in Patients With HBV and HDV Co-Infection","status":"UNKNOWN","sponsor":"National Taiwan University Hospital","startDate":"2023-02-24","conditions":["Hepatitis D"],"enrollment":30,"completionDate":"2025-08-31"},{"nctId":"NCT04910178","phase":"PHASE4","title":"Follow-up of NAFLD Patients With MRI-PDFF","status":"COMPLETED","sponsor":"Asmaa Abdelfattah Elsayed","startDate":"2020-12-01","conditions":["Diabetes Type 2","NAFLD"],"enrollment":80,"completionDate":"2021-12-30"},{"nctId":"NCT04810156","phase":"PHASE2","title":"Treatment Efficacy of Corticosteroids and Mycophenolate Mofetil in Patients With Immune Related Hepatitis","status":"UNKNOWN","sponsor":"Inge Marie Svane","startDate":"2021-04-07","conditions":["Hepatitis, Drug-Induced"],"enrollment":60,"completionDate":"2025-11-07"},{"nctId":"NCT05500937","phase":"PHASE2,PHASE3","title":"Effect of UDCA on the New Onset Diabetes and Glucose Intolerance Induced by Statin","status":"UNKNOWN","sponsor":"First Affiliated Hospital Xi'an Jiaotong University","startDate":"2021-03-01","conditions":["Diabetes Mellitus"],"enrollment":1000,"completionDate":"2024-12-31"},{"nctId":"NCT05499026","phase":"","title":"Safety and Efficacy of CDCA in CTX Chenodeoxycholic Acid (CDCA) in Patients Affected by Cerebrotendinous Xanthomatosis (CTX)","status":"COMPLETED","sponsor":"Leadiant Biosciences Ltd.","startDate":"2014-12-09","conditions":["Cerebrotendinous Xanthomatoses"],"enrollment":28,"completionDate":"2015-06-03"},{"nctId":"NCT03726229","phase":"","title":"Cholate Clearance in Fontans","status":"COMPLETED","sponsor":"University of Pennsylvania","startDate":"2019-05-01","conditions":["Fontan Procedure"],"enrollment":50,"completionDate":"2022-05-25"},{"nctId":"NCT01589523","phase":"PHASE3","title":"GlycoCholic Acid Treatment for Patients With Inborn Errors in Bile Acid Synthesis","status":"COMPLETED","sponsor":"Children's Hospital Medical Center, Cincinnati","startDate":"2006-02","conditions":["Bile Acid Synthesis Defect","Inborn Error of Bile Acid Metabolism","Inborn Error of Bile Acid Conjugation"],"enrollment":5,"completionDate":"2019-01-22"},{"nctId":"NCT03168555","phase":"PHASE4","title":"Changes in Bile Acid Homeostasis and Stool Habits After Cholecystectomy","status":"COMPLETED","sponsor":"Zealand University Hospital","startDate":"2017-06-22","conditions":["Bile Acid Malabsorption","Cholelithiasis"],"enrollment":23,"completionDate":"2019-05-15"},{"nctId":"NCT02967250","phase":"PHASE1","title":"Brain Bioenergetics in Parkinson's Disease and Response to Repeated Oral UDCA Treatment","status":"COMPLETED","sponsor":"University of Minnesota","startDate":"2020-04-01","conditions":["Parkinson Disease"],"enrollment":5,"completionDate":"2022-02-28"},{"nctId":"NCT03844100","phase":"PHASE1,PHASE2","title":"Effectiveness and Safety in Administrating CNU® Capsule to Refractory Functional Dyspepsia Patients","status":"COMPLETED","sponsor":"Gangnam Severance Hospital","startDate":"2019-11-01","conditions":["Dyspepsia","Biliary Dysplasia"],"enrollment":37,"completionDate":"2021-08-04"},{"nctId":"NCT02748616","phase":"PHASE4","title":"C. Difficile and Ursodiol","status":"COMPLETED","sponsor":"NYU Langone Health","startDate":"2017-03-01","conditions":["Diarrhea"],"enrollment":9,"completionDate":"2020-01-31"},{"nctId":"NCT03510598","phase":"PHASE4","title":"Submental Study (Sequential Treatment Approach)","status":"COMPLETED","sponsor":"Zeltiq Aesthetics","startDate":"2018-03-27","conditions":["Body Fat Disorder"],"enrollment":16,"completionDate":"2018-12-08"},{"nctId":"NCT02936596","phase":"NA","title":"Remission Induction of Primary Biliary Cholangitis-autoimmune Hepatitis Overlap Syndrome","status":"UNKNOWN","sponsor":"Xiaoli Fan","startDate":"2016-12","conditions":["Hepatitis, Autoimmune","Cholangitis","Liver Cirrhosis, Biliary","Cholestasis"],"enrollment":53,"completionDate":"2022-06-01"},{"nctId":"NCT02021110","phase":"PHASE2","title":"Ursodeoxycholic Acid as Treatment for Polycystic Liver Disease","status":"COMPLETED","sponsor":"Radboud University Medical Center","startDate":"2013-12","conditions":["Polycystic Liver Disease","Polycystic Kidney, Autosomal Dominant"],"enrollment":34,"completionDate":"2015-10"},{"nctId":"NCT05043194","phase":"NA","title":"Ursodeoxycholic Acid Prevents Total Parenteral Nutrition Cholestasis","status":"UNKNOWN","sponsor":"Wei Liu","startDate":"2021-01-01","conditions":["Cholestasis of Parenteral Nutrition"],"enrollment":80,"completionDate":"2022-07-30"},{"nctId":"NCT02965911","phase":"PHASE1,PHASE2","title":"Efficacy and Safety of Fenofibrate Combined With UDCA in PBC Patients With an Incomplete Biochemical Response to UDCA","status":"WITHDRAWN","sponsor":"Beijing 302 Hospital","startDate":"2016-01","conditions":["Primary Biliary Cirrhosis"],"enrollment":0,"completionDate":"2021-09-08"},{"nctId":"NCT04281082","phase":"","title":"Genetic Polymorphisms of ABCB11 and ABCB4 in Women With Intrahepatic Cholestasis of Pregnancy (ICP) and in Their First Degree Relatives","status":"UNKNOWN","sponsor":"Alexandra Hospital, Athens, Greece","startDate":"2018-01-01","conditions":["Cholestasis of Pregnancy"],"enrollment":50,"completionDate":"2022-01-31"},{"nctId":"NCT03087968","phase":"EARLY_PHASE1","title":"Evaluation of HepQuant SHUNT to Assess Liver Disease; Substudy Within GS-US-416-2124","status":"WITHDRAWN","sponsor":"HepQuant, LLC","startDate":"2016-07-31","conditions":["Severe Alcoholic Hepatitis"],"enrollment":0,"completionDate":"2017-09-13"},{"nctId":"NCT03294941","phase":"PHASE2","title":"The HepQuant SHUNT Test for Monitoring Liver Disease and Treatment Effects by Measuring Liver Function and Physiology","status":"TERMINATED","sponsor":"HepQuant, LLC","startDate":"2017-12-08","conditions":["NASH - Nonalcoholic Steatohepatitis"],"enrollment":17,"completionDate":"2019-09-11"},{"nctId":"NCT02523196","phase":"PHASE3","title":"Correlation and Comparison of the HepQuant® Disease Severity Index (DSI) With Hepatic Venous Pressure Gradient (HVPG)","status":"COMPLETED","sponsor":"University of Colorado, Denver","startDate":"2016-03","conditions":["Liver Disease"],"enrollment":20,"completionDate":"2017-02-23"},{"nctId":"NCT04977661","phase":"PHASE4","title":"Comparing the Effects of Vitamin E, Ursodeoxycholic Acid and Pentoxyfylline on Egyptian Non-alcoholic Steatohepatitis (NASH) Patients","status":"COMPLETED","sponsor":"Kafrelsheikh University","startDate":"2020-02-01","conditions":["Nonalcoholic Steatohepatitis (NASH)"],"enrollment":102,"completionDate":"2021-01-10"},{"nctId":"NCT03294850","phase":"NA","title":"Biomarkers of Liver Pathology in Patients With Presumed Non-Alcoholic Steatohepatitis Following Bariatric Surgery","status":"COMPLETED","sponsor":"AdventHealth Translational Research Institute","startDate":"2018-07-18","conditions":["NASH - Nonalcoholic Steatohepatitis; NAFLD - Nonalcoholic Fatty Liver Disease"],"enrollment":14,"completionDate":"2020-09-11"},{"nctId":"NCT00059202","phase":"PHASE2,PHASE3","title":"Trial of High-dose Urso in Primary Sclerosing Cholangitis","status":"TERMINATED","sponsor":"National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)","startDate":"2002-07","conditions":["Primary Sclerosing Cholangitis"],"enrollment":150,"completionDate":"2008-06"},{"nctId":"NCT03201952","phase":"PHASE1","title":"Examination of the Metabolic Effects of Direct Bile Salt Delivery to the Ileum in Humans","status":"TERMINATED","sponsor":"Vanderbilt University Medical Center","startDate":"2018-02-01","conditions":["Glucose Intolerance","Gastric Bypass Surgery"],"enrollment":2,"completionDate":"2020-05-01"},{"nctId":"NCT04089722","phase":"PHASE4","title":"Efficacy and Safety of Deoxycholic Acid Injections for Removal of Adipose Tissue in the \"Bra Strap Fat\" Region","status":"COMPLETED","sponsor":"Juva Skin & Laser Center","startDate":"2019-07-26","conditions":["Brassiere Strap Fat (BSF)","Bra Strap Fat (BSF)"],"enrollment":22,"completionDate":"2020-10-22"}],"_emaApprovals":[{"date":"2013-09-12","status":"Authorised","company":"Laboratoires CTRS"}],"genericFilers":[],"latestUpdates":[],"manufacturing":[],"administration":{"route":"Oral","formulation":"Capsule","formulations":[{"form":"CAPSULE","route":"ORAL","productName":"Cholbam"}]},"_patentsChecked":true,"crossReferences":{"NUI":"N0000191551","MMSL":"233415","NDDF":"007568","UNII":"G1JO7801AE","VUID":"4034356","CHEBI":"CHEBI:16359","VANDF":"4034356","RXNORM":"1440856","UMLSCUI":"C0055568","chemblId":"CHEMBL205596","ChEMBL_ID":"CHEMBL205596","DRUGBANK_ID":"DB02659","PDB_CHEM_ID":" CHD","PUBCHEM_CID":"221493","SNOMEDCT_US":"17147002","IUPHAR_LIGAND_ID":"609","MESH_DESCRIPTOR_UI":"D019826"},"formularyStatus":[],"_enricherVersion":"v2","developmentCodes":[],"ownershipHistory":[{"period":"2015-","companyName":"Rtrx","relationship":"Original Developer"},{"period":"present","companyName":"Mirum","relationship":"Current Owner"},{"period":"2013","companyName":"Laboratoires CTRS","relationship":"EMA Licensee"},{"period":"2023","companyName":"ReqMed Company, Ltd","relationship":"PMDA Licensee"}],"publicationCount":4531,"therapeuticAreas":["Metabolic"],"atcClassification":{"source":"DrugCentral","atcCode":"A05AA03","allCodes":["A05AA03"]},"biosimilarFilings":[],"originalDeveloper":"Rtrx","recentPublications":[{"date":"2026 Mar 25","pmid":"41895615","title":"Altered serum 3β-hydroxy bile acids in major depressive disorder and associations with symptom dimensions.","journal":"Journal of affective disorders"},{"date":"2026 Mar 24","pmid":"41887383","title":"The mechanism of Tibetan medicine Shibawei Niuhuang Qinggan pills in protecting against acetaminophen-induced acute liver injury in mice by regulating the Keap1-Nrf2 pathway.","journal":"Journal of ethnopharmacology"},{"date":"2026 Mar 24","pmid":"41880860","title":"Usefulness of plasma bile acid profile as a prognostic biomarker for drug-induced liver injury.","journal":"EBioMedicine"},{"date":"2026 Mar 23","pmid":"41869696","title":"Bilberry (Vaccinium myrtillus L.) extract alleviates gestational diabetes mellitus by coordinating TGR5/FXR signaling via a gut microbiota-bile acid axis.","journal":"Food & function"},{"date":"2026 Jun","pmid":"41859732","title":"Autosomal recessive ELOVL1-related disorder presenting with severe neonatal cholestasis: A novel clinical feature?","journal":"Molecular genetics and metabolism reports"}],"companionDiagnostics":[],"genericManufacturers":0,"_genericFilersChecked":true,"genericManufacturerList":[],"status":"approved","companyName":"Mirum","companyId":"mirum","modality":"Small molecule","firstApprovalDate":"2015","enrichmentLevel":4,"visitCount":0,"regulatoryByCountry":[{"country_code":"US","regulator":"FDA","status":"approved","approval_date":"2015-03-17T00:00:00.000Z","mah":"RTRX","brand_name_local":null,"application_number":""},{"country_code":"IL","regulator":"MOH","status":"likely_approved","approval_date":"2015-03-17T00:00:00.000Z","mah":"RTRX","brand_name_local":null,"application_number":null},{"country_code":"SG","regulator":"HSA","status":"likely_approved","approval_date":"2015-03-17T00:00:00.000Z","mah":"RTRX","brand_name_local":null,"application_number":null},{"country_code":"SA","regulator":"SFDA","status":"likely_approved","approval_date":"2015-03-17T00:00:00.000Z","mah":"RTRX","brand_name_local":null,"application_number":null},{"country_code":"AE","regulator":"MOH","status":"likely_approved","approval_date":"2015-03-17T00:00:00.000Z","mah":"RTRX","brand_name_local":null,"application_number":null},{"country_code":"KR","regulator":"MFDS","status":"likely_approved","approval_date":"2015-03-17T00:00:00.000Z","mah":"RTRX","brand_name_local":null,"application_number":null},{"country_code":"US","regulator":"FDA","status":"approved","approval_date":"2015-03-17T00:00:00.000Z","mah":"MIRUM","brand_name_local":null,"application_number":"NDA205750"},{"country_code":"JP","regulator":"PMDA","status":"approved","approval_date":"2023-03-27T00:00:00.000Z","mah":"ReqMed Company, Ltd","brand_name_local":null,"application_number":""},{"country_code":"TH","regulator":"FDA-TH","status":"likely_approved","approval_date":null,"mah":null,"brand_name_local":null,"application_number":null},{"country_code":"MY","regulator":"NPRA","status":"likely_approved","approval_date":null,"mah":null,"brand_name_local":null,"application_number":null},{"country_code":"PH","regulator":"FDA-PH","status":"likely_approved","approval_date":null,"mah":null,"brand_name_local":null,"application_number":null},{"country_code":"CO","regulator":"INVIMA","status":"likely_approved","approval_date":null,"mah":null,"brand_name_local":null,"application_number":null},{"country_code":"ZA","regulator":"SAHPRA","status":"likely_approved","approval_date":null,"mah":null,"brand_name_local":null,"application_number":null},{"country_code":"TW","regulator":"TFDA","status":"likely_approved","approval_date":null,"mah":null,"brand_name_local":null,"application_number":null},{"country_code":"HK","regulator":"DH","status":"likely_approved","approval_date":null,"mah":null,"brand_name_local":null,"application_number":null},{"country_code":"AU","regulator":"TGA","status":"likely_approved","approval_date":null,"mah":null,"brand_name_local":null,"application_number":null},{"country_code":"AU","regulator":"TGA","status":"likely_approved","approval_date":null,"mah":null,"brand_name_local":null,"application_number":null},{"country_code":"GB","regulator":"MHRA","status":"likely_approved","approval_date":null,"mah":null,"brand_name_local":null,"application_number":null},{"country_code":"GB","regulator":"MHRA","status":"likely_approved","approval_date":null,"mah":null,"brand_name_local":null,"application_number":null},{"country_code":"EU","regulator":"EMA","status":"pending","approval_date":null,"mah":"","brand_name_local":"","application_number":""},{"country_code":"CA","regulator":"Health Canada","status":"approved","approval_date":null,"mah":"","brand_name_local":"","application_number":""},{"country_code":"BR","regulator":"ANVISA","status":"likely_approved","approval_date":null,"mah":null,"brand_name_local":null,"application_number":null},{"country_code":"MX","regulator":"COFEPRIS","status":"likely_approved","approval_date":null,"mah":null,"brand_name_local":null,"application_number":null},{"country_code":"AR","regulator":"ANMAT","status":"likely_approved","approval_date":null,"mah":null,"brand_name_local":null,"application_number":null},{"country_code":"TR","regulator":"TITCK","status":"likely_approved","approval_date":null,"mah":null,"brand_name_local":null,"application_number":null},{"country_code":"IN","regulator":"CDSCO","status":"likely_approved","approval_date":null,"mah":null,"brand_name_local":null,"application_number":null}],"trialStats":{"total":0,"withResults":0},"validation":{"fieldsValidated":1,"lastValidatedAt":"2026-04-20T00:42:11.779630+00:00","fieldsConflicting":0,"overallConfidence":0.95},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}