{"id":"chenodiol","rwe":[{"pmid":"41324091","year":"2025","title":"FDA Approves First Targeted Treatment for Cerebrotendinous Xanthomatosis: A Perspective on a Landmark in Rare Lipid Storage Disease Therapy.","finding":"","journal":"Health science reports","studyType":"Clinical Study"},{"pmid":"40864842","year":"2006","title":"Chenodiol.","finding":"","journal":"","studyType":"Clinical Study"},{"pmid":"40443237","year":"2025","title":"Regulatory News: Chenodiol for the Treatment of Cerebrotendinous Xanthomatosis: FDA Approval Summary.","finding":"","journal":"Journal of inherited metabolic disease","studyType":"Clinical Study"},{"pmid":"40185658","year":"2025","title":"CTEXLI (chenodiol).","finding":"","journal":"Clinical therapeutics","studyType":"Clinical Study"},{"pmid":"35899436","year":"2022","title":"Differential effects of metformin-mediated BSEP repression on pravastatin and bile acid pharmacokinetics in humans: A randomized controlled trial.","finding":"","journal":"Clinical and translational science","studyType":"Clinical Study"}],"_fda":{"id":"71b050b3-07a6-401f-9526-104383bff7b7","set_id":"015384e7-4ac5-4782-a309-e40cd1421c5b","openfda":{"nui":["N0000175802","M0002475"],"unii":["0GEI24LG0J"],"route":["ORAL"],"rxcui":["618469","2710422"],"spl_id":["71b050b3-07a6-401f-9526-104383bff7b7"],"brand_name":["CTEXLI"],"spl_set_id":["015384e7-4ac5-4782-a309-e40cd1421c5b"],"package_ndc":["79378-310-90"],"product_ndc":["79378-310"],"generic_name":["CHENODIOL"],"product_type":["HUMAN PRESCRIPTION DRUG"],"pharm_class_cs":["Bile Acids and Salts [CS]"],"substance_name":["CHENODIOL"],"pharm_class_epc":["Bile Acid [EPC]"],"manufacturer_name":["Mirum Pharmaceuticals Inc."],"application_number":["NDA219488"],"is_original_packager":[true]},"version":"2","pregnancy":["8.1 Pregnancy Risk Summary Available data from published case reports over decades of use with chenodiol during pregnancy have not identified an increased risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes. Serious hepatic, renal and adrenal lesions occurred in fetuses of female Rhesus monkeys treated at doses 1 to 2 times the recommended human dose based on body surface area (mg/m 2 ). Hepatic lesions also occurred at doses comparable to the human dose based on body surface area in neonatal baboons born to mothers administered chenodiol during pregnancy ( see Data ). The animal study findings have not been demonstrated with human use. The background risk of major birth defects and miscarriage for the indicated population is unknown. All pregnancies have a background risk of birth defect, loss, or other adverse outcomes. In the US general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2 to 4% and 15 to 20%, respectively. Data Animal Data Hepatic lesions were reported in neonatal baboons whose mothers had received 18 to 38 mg/kg of chenodiol throughout pregnancy (0.6 to 1.4 times the recommended human dose based on body surface area). Serious hepatic, renal and adrenal lesions were also reported in fetuses of female Rhesus monkeys given 60 to 90 mg/kg/day from GD 21-45 of pregnancy (1 to 2 times the recommended human dose based on body surface area). Non-human primates form sulfate conjugates of the known hepatotoxic bacterial metabolite of chenodiol, lithocholic acid, to a lesser extent than reported in humans, which may exaggerate the toxicity of orally dosed chenodiol compared to humans. However, there is also evidence that the hepatobiliary toxicity is partly due to the parent drug, chenodiol."],"overdosage":["10 OVERDOSAGE Cases of intentional overdose with chenodiol have been reported: one patient consumed 3 g to 4.5 g of chenodiol and another patient consumed 30 g of chenodiol. Clinical manifestations experienced by these patients included nausea, dizziness, and diarrhea. In the event of an overdose, discontinue CTEXLI, monitor the patient, and institute general supportive measures if needed."],"description":["11 DESCRIPTION CTEXLI (chenodiol) is a bile acid. Chenodiol is a bitter-tasting, white powder consisting of crystalline and amorphous particles that are freely soluble in methanol, acetone and acetic acid, and practically insoluble in water. The chemical name of chenodiol is 3α,7α-dihydroxy-5-β-cholan-24-oic acid. The molecular formula is C 24 H 40 O 4 and the molecular weight is 392.58 g/mol. The chemical structure is: Each CTEXLI tablet contains 250 mg of chenodiol. Inactive ingredients are magnesium stearate, microcrystalline cellulose, pregelatinized starch, silicon dioxide, and sodium starch glycolate. The thin-film coating contains opadry YS 2 7035 (consisting of methylcellulose and glycerin) and sodium lauryl sulfate. Chenodiol chemical structure"],"how_supplied":["16 HOW SUPPLIED/STORAGE AND HANDLING How Supplied CTEXLI (chenodiol) tablets are supplied as 250 mg white film-coated tablets imprinted with “MP” on one side and \"250\" on the other side. NDC 79378-310-90: 100 count bottle Storage and Handling Store CTEXLI at 20°C to 25°C (68°F to 77°F); excursions permitted between 15°C to 30°C (59°F to 86°F) [see USP Controlled Room Temperature]."],"geriatric_use":["8.5 Geriatric Use Trial 1 of CTEXLI in patients with CTX did not include patients aged 65 years and older."],"pediatric_use":["8.4 Pediatric Use The safety and effectiveness of CTEXLI for the treatment of CTX have not been established in pediatric patients."],"effective_time":"20251216","clinical_studies":["14 CLINICAL STUDIES The efficacy of CTEXLI for the treatment of patients with CTX was evaluated in Trial 1, which was a randomized, double-blind, placebo controlled, 2-period with 2-treatment crossover trial in patients ≥16 years of age (NCT 04270682). In Trial 1, 14 patients were enrolled and 13 patients were randomized and treated in a crossover withdrawal design to receive either CTEXLI 250 mg or placebo orally three times daily for 4 weeks during 2 double-blind treatment periods. The study also included treatment with CTEXLI 250 mg three times daily during an 8 week run-in period and an 8-week open label period in between the 2 double-blind withdrawal periods. The total duration of study treatment was 24 weeks. Of the 13 randomized patients, 62% were male and 39% were female. The baseline median age was 42 years (16-55) and median age at diagnosis was 35 years (15-55). CTEXLI is not approved for use in pediatric patients. The patient population consisted of 62% White, 15% Asian, and 23% Other (In the Other racial group, there was one patient who reported both White and Black). Ethnicity consisted of 15% Hispanic or Latino, 54% not Hispanic or Latino, and 31% unknown. Plasma cholestanol and urine 23S-pentol were assessed at multiple time points as shown in Figure 1 . For plasma cholestanol, the estimated mean change from baseline at day 29 was -2.3 µg/mL when patients continued CTEXLI treatment and 6.2 µg/mL when patients received placebo. The estimated treatment difference was -8.5 µg/mL (95% CI: -13.2, -3.9) ( Table 1 ). For urine 23S-pentol, the estimated mean change from baseline at day 29 was 185 ng/mL when patients continued CTEXLI treatment and 29506 ng/mL when patients received placebo. The estimated treatment difference was -29321 ng/mL (95% CI: -45701, -12941). Figure 1: Mean (SE) of Observed Plasma Cholestanol by Treatment Sequence (All Randomized Patients) DB = double-blind. Solid line represents treatment with CTEXLI and dashed line represents treatment with placebo. The mean values of plasma cholestanol at baselines and end of each DB period are annotated in the figure. Table 1: Summary Results for Plasma Cholestanol and Urine 23S Pentol For each study treatment (placebo or CTEXLI), the mean value at Baseline was calculated as the mean of the measurements obtained prior to receiving the study treatment during the double-blind study duration; and the mean value at Day 29 was calculated as the mean of the measurements at Day 29 at the end of the study treatment. For each patient at each visit, the measurement of urine 23S-pentol was calculated as the geometric mean of first 3 morning void urine samples collected within 5 days prior to the visit. Plasma Cholestanol (µg/mL) Mean (SD) CTEXLI (N = 13) Placebo (N = 13) Baseline 10.8 (10.0) 8.8 (7.8) Day 29 8.5 (7.0) 15.1 (8.8) Change from Baseline at Day 29 -2.3 (3.9) 6.2 (5.6) Treatment Difference -8.5 (95% CI: -13.2, -3.9) Urine 23S- Pentol (ng/mL) Baseline 1811 (1693) 1773 (1940) Day 29 1996 (1341) 31279 (27595) Change from Baseline at Day 29 185 (1479) 29506 (27257) Treatment Difference -29321 (95% CI: -45701, -12941) Figure 1: Mean (SE) of Observed Plasma Cholestanol by Treatment Sequence (All Randomized Patients)"],"pharmacodynamics":["12.2 Pharmacodynamics In Trial 1, plasma cholestanol and urine 23S-pentol concentrations were elevated in patients with CTX. Treatment with CTEXLI resulted in reductions of plasma cholestanol and urine 23S-pentol concentrations in the 8-week run-in open label treatment period. Continued treatment with CTEXLI for 4 weeks in the double-blind treatment period resulted in the maintenance of low levels of urine 23S-pentol and additional reductions of plasma cholestanol [see Clinical Studies ( 14 )] ."],"pharmacokinetics":["12.3 Pharmacokinetics In CTX patients, the geometric mean (%CV) maximum plasma concentration (C max ), trough plasma concentration (C trough ), and area under the plasma concentration-time curve (AUC 0-8h ) of chenodiol at steady state following the recommended dosage (250 mg administered orally three times daily) were 3.7 mcg/mL (60%), 0.7 mcg/mL (90%), and 12.5 mcg*h/mL (60%), respectively. Absorption The median (range) T max of chenodiol following an oral administration in CTX patients was 3 (0.5‑8) hours. Distribution Due to first-pass hepatic clearance, the body pool of chenodiol resides mainly in the enterohepatic circulation. The apparent volume of distribution of chenodiol at steady-state was 0.36 L/kg. The plasma protein binding of chenodiol was approximately 98%. Elimination The geometric mean total apparent clearance of chenodiol in CTX patients was 20 L/h. Metabolism Chenodiol is well absorbed from the small intestine and taken up by the liver where it is converted to its taurine and glycine conjugates and secreted into the bile along with other endogenous bile acids in the enterohepatic circulation. Chenodiol that escapes to the colon is converted by bacterial action to lithocholic acid. Humans have the capacity to form sulfate conjugates of lithocholic acid. About 80% of the lithocholate is excreted in the feces and the remainder is absorbed and converted in the liver to its poorly absorbed sulfolithocholyl conjugates. Excretion Conjugated chenodiol is either reabsorbed in the terminal ileum, deconjugated before excretion, or decomposed by bacteria to lithocholic acid. Drug Interaction Studies Based on in vitro studies, chenodiol and its glyco- and tauro- conjugates are not expected to inhibit CYPs 1A2, 2B6, 2C8, 2C9, 2C19, 2D6, or 3A4, or induce CYPs 1A2 or 2B6 at the recommended dose of chenodiol of 250 mg TID. Chenodiol and its tauro- conjugate may upregulate CYP3A4 mRNA in vitro. The clinical significance of this upregulation is unknown. The glyco- and tauro- conjugates of chenodiol are high affinity substrates for BSEP, and in vitro studies suggest that chenodiol may inhibit OATP1B1 and OATP1B3 at the recommended dose of 250 mg TID (clinical significance unknown), but chenodiol and its glyco- and tauro- conjugates are not predicted to inhibit P-gp, BCRP, OATP2B1, OAT1, OAT3, OCT1, OCT2, MATE1, or MATE2-K."],"adverse_reactions":["6 ADVERSE REACTIONS The following clinically significant adverse reaction is described elsewhere in the labeling: • Hepatotoxicity [see Warnings and Precautions ( 5.1 )] The most common adverse reactions (incidence > 14%) are diarrhea, headache, abdominal pain, constipation, hypertension, muscular weakness, and upper respiratory tract infection. ( 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. The safety of CTEXLI was evaluated in a randomized, double blind, placebo-controlled, 2-period, 2-treatment crossover trial in 14 patients (16 to 55 years of age) with CTX (Trial 1). CTEXLI is not approved for use in pediatric patients. The dosage of CTEXLI was 250 mg orally three times a day [see Clinical Studies ( 14 )]. The mean (SD) chenodiol exposure during Trial 1 was 139.1 (26.7) days. The most common adverse reactions which occurred in two or more patients ( > 14%) during CTEXLI treatment (including the two 8-week open-label treatment periods) were diarrhea (36%), headache (21%), and abdominal pain (including abdominal pain upper) (14%), constipation (14%), hypertension (14%), muscular weakness (14%), and upper respiratory tract infection (14%). In Trial 1, one CTEXLI-treated patient (7%) had increased ALT levels > 3x ULN, which led to treatment interruption. 6.2 Postmarketing Experience The following adverse reactions have been identified during post approval use of chenodiol. Because these reactions are reported voluntarily from a population of uncertain size, it is not always possible to reliably estimate their frequency or establish a causal relationship to drug exposure. • Hepatobiliary Disorders: Hepatotoxicity [see Warnings and Precautions ( 5.1 )] • Immune System Disorders: Hypersensitivity reactions such as facial swelling, pruritus, rash, urticaria."],"contraindications":["4 CONTRAINDICATIONS None. None. ( 4 )"],"drug_interactions":["7 DRUG INTERACTIONS • Bile acid sequestering agents and aluminum-based antacids: Avoid concomitant use with CTEXLI. ( 7.1 ) • Coumarin and its derivatives: Monitor prothrombin time and adjust dosage accordingly. ( 7.2 ) 7.1 Effect of Other Drugs on CTEXLI Co-administration of bile acid sequestering agents, such as cholestyramine and colestipol, or aluminum-based antacids may decrease absorption of CTEXLI in the intestine and may result in decreased efficacy of CTEXLI. Avoid concomitant use of bile acid sequestering agents or aluminum-based antacids with CTEXLI. 7.2 Effect of CTEXLI on Other Drugs Due to potential hepatotoxicity, CTEXLI may affect the pharmacodynamics of coumarin and its derivatives, causing unexpected prolongation of the prothrombin time and hemorrhage. If concomitant use of CTEXLI with coumarin or its derivatives is unavoidable, monitor prothrombin time. Adjust the dosage of coumarin or its derivatives in accordance with its approved product labeling."],"mechanism_of_action":["12.1 Mechanism of Action Endogenous chenodiol (chenodeoxycholic acid) is a primary bile acid, synthesized from cholesterol in the liver. In CTX, the major bile acid synthesis pathways are disrupted due to partial or total deficiency in sterol 27-hydroxylase encoded by the CYP27A1 gene. CTEXLI may act to replace deficient levels of the endogenous bile acid chenodeoxycholic acid in patients with CTX. Increased chenodiol levels in the enterohepatic bile acid pool restore the activation of farnesoid X receptor (FXR) and downregulate CYP7A1 leading to suppression and reduction of atypical bile acids and bile alcohols including cholestanol and 23S-pentol."],"clinical_pharmacology":["12 CLINICAL PHARMACOLOGY 12.1 Mechanism of Action Endogenous chenodiol (chenodeoxycholic acid) is a primary bile acid, synthesized from cholesterol in the liver. In CTX, the major bile acid synthesis pathways are disrupted due to partial or total deficiency in sterol 27-hydroxylase encoded by the CYP27A1 gene. CTEXLI may act to replace deficient levels of the endogenous bile acid chenodeoxycholic acid in patients with CTX. Increased chenodiol levels in the enterohepatic bile acid pool restore the activation of farnesoid X receptor (FXR) and downregulate CYP7A1 leading to suppression and reduction of atypical bile acids and bile alcohols including cholestanol and 23S-pentol. 12.2 Pharmacodynamics In Trial 1, plasma cholestanol and urine 23S-pentol concentrations were elevated in patients with CTX. Treatment with CTEXLI resulted in reductions of plasma cholestanol and urine 23S-pentol concentrations in the 8-week run-in open label treatment period. Continued treatment with CTEXLI for 4 weeks in the double-blind treatment period resulted in the maintenance of low levels of urine 23S-pentol and additional reductions of plasma cholestanol [see Clinical Studies ( 14 )] . 12.3 Pharmacokinetics In CTX patients, the geometric mean (%CV) maximum plasma concentration (C max ), trough plasma concentration (C trough ), and area under the plasma concentration-time curve (AUC 0-8h ) of chenodiol at steady state following the recommended dosage (250 mg administered orally three times daily) were 3.7 mcg/mL (60%), 0.7 mcg/mL (90%), and 12.5 mcg*h/mL (60%), respectively. Absorption The median (range) T max of chenodiol following an oral administration in CTX patients was 3 (0.5‑8) hours. Distribution Due to first-pass hepatic clearance, the body pool of chenodiol resides mainly in the enterohepatic circulation. The apparent volume of distribution of chenodiol at steady-state was 0.36 L/kg. The plasma protein binding of chenodiol was approximately 98%. Elimination The geometric mean total apparent clearance of chenodiol in CTX patients was 20 L/h. Metabolism Chenodiol is well absorbed from the small intestine and taken up by the liver where it is converted to its taurine and glycine conjugates and secreted into the bile along with other endogenous bile acids in the enterohepatic circulation. Chenodiol that escapes to the colon is converted by bacterial action to lithocholic acid. Humans have the capacity to form sulfate conjugates of lithocholic acid. About 80% of the lithocholate is excreted in the feces and the remainder is absorbed and converted in the liver to its poorly absorbed sulfolithocholyl conjugates. Excretion Conjugated chenodiol is either reabsorbed in the terminal ileum, deconjugated before excretion, or decomposed by bacteria to lithocholic acid. Drug Interaction Studies Based on in vitro studies, chenodiol and its glyco- and tauro- conjugates are not expected to inhibit CYPs 1A2, 2B6, 2C8, 2C9, 2C19, 2D6, or 3A4, or induce CYPs 1A2 or 2B6 at the recommended dose of chenodiol of 250 mg TID. Chenodiol and its tauro- conjugate may upregulate CYP3A4 mRNA in vitro. The clinical significance of this upregulation is unknown. The glyco- and tauro- conjugates of chenodiol are high affinity substrates for BSEP, and in vitro studies suggest that chenodiol may inhibit OATP1B1 and OATP1B3 at the recommended dose of 250 mg TID (clinical significance unknown), but chenodiol and its glyco- and tauro- conjugates are not predicted to inhibit P-gp, BCRP, OATP2B1, OAT1, OAT3, OCT1, OCT2, MATE1, or MATE2-K."],"indications_and_usage":["1 INDICATIONS AND USAGE CTEXLI is indicated for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. CTEXLI is a bile acid indicated for treatment of cerebrotendinous xanthomatosis (CTX) in adults. ( 1 )"],"warnings_and_cautions":["5 WARNINGS AND PRECAUTIONS Hepatotoxicity : Obtain baseline liver transaminase and total bilirubin levels in all patients and monitor yearly and as clinically indicated. Interrupt treatment until the levels have returned to baseline values. For persistent or recurrent liver test abnormalities, consider discontinuing CTEXLI. ( 5.1 ) 5.1 Hepatotoxicity Chenodiol, including CTEXLI, has been associated with hepatotoxicity [see Adverse Reactions ( 6 )] . In Trial 1, one CTEXLI-treated patient (7%) had increased ALT levels > 3 times ULN, which led to treatment interruption. Patients with pre-existing liver disease or bile duct abnormalities may be at higher risk for hepatotoxicity during treatment with CTEXLI. Published reports suggest patients who are poor sulfators of lithocholic acid are more likely to develop chenodiol-induced serum aminotransferase elevations [see Clinical Pharmacology ( 12.3 )] . Obtain baseline liver transaminase (ALT, AST) and total bilirubin levels in all patients prior to treatment initiation with CTEXLI. If liver transaminase levels are elevated > 3 times ULN or total bilirubin level is >2 times ULN, interrupt treatment with CTEXLI until the levels have returned to baseline values. Monitor liver transaminase and total bilirubin levels yearly and as clinically indicated. For persistent or recurrent liver test abnormalities, consider discontinuing CTEXLI. Inform the patient of the symptoms of hepatotoxicity (e.g., abdominal pain, bruising, dark-colored urine, fatigue, bleeding, jaundice, nausea, and pruritus). If clinical signs and symptoms consistent with hepatotoxicity occur, have the patient discontinue CTEXLI immediately."],"clinical_studies_table":["<table ID=\"_Ref191385162\" width=\"100%\"><caption>Table 1: Summary Results for Plasma Cholestanol and Urine 23S Pentol</caption><col width=\"15%\"/><col width=\"40%\"/><col width=\"22%\"/><col width=\"23%\"/><tfoot><tr><td align=\"left\" colspan=\"4\" valign=\"top\">For each study treatment (placebo or CTEXLI), the mean value at Baseline was calculated as the mean of the measurements obtained prior to receiving the study treatment during the double-blind study duration; and the mean value at Day 29 was calculated as the mean of the measurements at Day 29 at the end of the study treatment. For each patient at each visit, the measurement of urine 23S-pentol was calculated as the geometric mean of first 3 morning void urine samples collected within 5 days prior to the visit.</td></tr></tfoot><tbody><tr><td rowspan=\"5\" styleCode=\"Rrule Botrule Lrule Toprule \" valign=\"middle\"><paragraph><content styleCode=\"bold\">Plasma Cholestanol (&#xB5;g/mL)</content></paragraph></td><td styleCode=\"Rrule Botrule Lrule Toprule \" valign=\"middle\"><paragraph><content styleCode=\"bold\">Mean (SD)</content></paragraph></td><td align=\"center\" styleCode=\"Rrule Botrule Lrule Toprule \" valign=\"middle\"><paragraph><content styleCode=\"bold\">CTEXLI (N = 13)</content></paragraph></td><td align=\"center\" styleCode=\"Rrule Botrule Lrule Toprule \" valign=\"middle\"><paragraph><content styleCode=\"bold\">Placebo</content></paragraph><paragraph><content styleCode=\"bold\">(N = 13)</content></paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"middle\"><paragraph> Baseline</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"middle\"><paragraph>10.8 (10.0)</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"middle\"><paragraph>8.8 (7.8)</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"middle\"><paragraph> Day 29</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"middle\"><paragraph>8.5 (7.0)</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"middle\"><paragraph>15.1 (8.8)</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"middle\"><paragraph> Change from Baseline at Day 29</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"middle\"><paragraph>-2.3 (3.9)</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"middle\"><paragraph>6.2 (5.6)</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"middle\"><paragraph> Treatment Difference</paragraph></td><td align=\"center\" colspan=\"2\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"middle\"><paragraph>-8.5 (95% CI: -13.2, -3.9)</paragraph></td></tr><tr><td rowspan=\"4\" styleCode=\"Rrule Lrule Botrule \" valign=\"middle\"><paragraph><content styleCode=\"bold\">Urine 23S- Pentol (ng/mL)</content></paragraph></td><td styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"middle\"><paragraph> Baseline</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"middle\"><paragraph>1811 (1693)</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"middle\"><paragraph>1773 (1940)</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"middle\"><paragraph> Day 29</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"middle\"><paragraph>1996 (1341)</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"middle\"><paragraph>31279 (27595)</paragraph></td></tr><tr><td styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"middle\"><paragraph> Change from Baseline at Day 29</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"middle\"><paragraph>185 (1479)</paragraph></td><td align=\"center\" styleCode=\"Rrule Lrule Toprule Botrule \" valign=\"middle\"><paragraph>29506 (27257)</paragraph></td></tr><tr><td styleCode=\"Rrule Botrule Lrule Toprule \" valign=\"middle\"><paragraph> Treatment Difference</paragraph></td><td align=\"center\" colspan=\"2\" styleCode=\"Rrule Botrule Lrule Toprule \" valign=\"middle\"><paragraph>-29321 (95% CI: -45701, -12941)</paragraph></td></tr></tbody></table>"],"nonclinical_toxicology":["13 NONCLINICAL TOXICOLOGY 13.1 Carcinogenesis, Mutagenesis, Impairment of Fertility A two-year oral study of chenodiol in rats did not show a carcinogenic potential at the tested levels of 15 to 60 mg/kg/day (0.2 to 0.6 times the recommended human dose based on body surface area). In additional long-term studies, chenodiol given at oral doses up to 600 mg/kg/day in rats (6 times the recommended human dose based on body surface area), and 1000 mg/kg/day in mice (5 times the recommended human dose based on body surface area) induced benign and malignant liver cell tumors in female rats and cholangiomas in female rats and male mice. Two-year studies of lithocholic acid (a major metabolite of chenodiol) in mice (125 to 250 mg/kg/day, equivalent to 0.7 to 1.4 times the recommended human dose based on body surface area) and rats (250 and 500 mg/kg/day, equivalent to 3 to 5 times the recommended human dose based on body surface area) found it not to be carcinogenic. The dietary administration of lithocholic acid to chickens is reported to cause hepatic adenomatous hyperplasia. 13.2 Animal Toxicology and/or Pharmacology Chenodiol caused hepatobiliary toxicity (e.g., cholestasis) in many animal species, including rodents, non-rodents, and non-human primates at doses close to the human dose. Less efficient sulfation of the chenodiol metabolite, lithocholic acid, in non-human primates compared to humans is thought to cause the hepatobiliary toxicity of orally dosed chenodiol. However, there is evidence that the hepatobiliary toxicity is partly due to the parent drug, chenodiol."],"information_for_patients":["17 PATIENT COUNSELING INFORMATION Hepatotoxicity Inform the patient of the symptoms of hepatotoxicity (e.g., abdominal pain, bruising, dark-colored urine, fatigue, bleeding, jaundice, nausea, and pruritus). Instruct the patient to discontinue CTEXLI immediately and seek medical care should symptoms occur [see Warnings and Precautions ( 5.1 )] . Rx only Manufactured for: Mirum Pharmaceuticals, Inc. Foster City, CA 94404 © 2025 Mirum Pharmaceuticals, Inc. CTEXLI ® is a registered trademark of Mirum Pharmaceuticals, Inc."],"dosage_and_administration":["2 DOSAGE AND ADMINISTRATION • Before initiating CTEXLI, obtain baseline liver transaminase (alanine aminotransferase [ALT] and aspartate aminotransferase [AST]) and total bilirubin levels in all patients. ( 2.1 ) • The recommended dosage is 250 mg orally three times daily. ( 2.2 ) 2.1 Important Recommendation Prior to CTEXLI Treatment Initiation Before initiating CTEXLI, obtain baseline liver transaminase (alanine aminotransferase [ALT] and aspartate aminotransferase [AST]) and total bilirubin levels in all patients [see Warnings and Precautions ( 5.1 )] . 2.2 Recommended Dosage The recommended dosage of CTEXLI is 250 mg administered orally three times daily. Administer CTEXLI with or without food. Swallow tablets whole. Missed Dose If a dose of CTEXLI is missed, advise the patient to skip the missed dose and to resume taking the prescribed dose at the next scheduled time. Patients should not take a double dose. 2.3 Administration Modification and Monitoring If liver transaminase (ALT, AST) levels are elevated > 3 times the upper limit of normal (ULN) or total bilirubin level is >2 times ULN, interrupt treatment with CTEXLI until the levels have returned to baseline values. Monitor liver transaminase and total bilirubin levels yearly and as clinically indicated [see Warnings and Precautions ( 5.1 )]."],"spl_product_data_elements":["CTEXLI chenodiol CHENODIOL CHENODIOL SILICON DIOXIDE MICROCRYSTALLINE CELLULOSE SODIUM STARCH GLYCOLATE TYPE A POTATO MAGNESIUM STEARATE METHYLCELLULOSE (100 MPA.S) GLYCERIN SODIUM LAURYL SULFATE STARCH, CORN MP;250"],"dosage_forms_and_strengths":["3 DOSAGE FORMS AND STRENGTHS Tablets: 250 mg of chenodiol as white film-coated tablets imprinted with “MP” on one side and \"250\" on the other side. CTEXLI tablets: 250 mg. ( 3 )"],"use_in_specific_populations":["8 USE IN SPECIFIC POPULATIONS 8.1 Pregnancy Risk Summary Available data from published case reports over decades of use with chenodiol during pregnancy have not identified an increased risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes. Serious hepatic, renal and adrenal lesions occurred in fetuses of female Rhesus monkeys treated at doses 1 to 2 times the recommended human dose based on body surface area (mg/m 2 ). Hepatic lesions also occurred at doses comparable to the human dose based on body surface area in neonatal baboons born to mothers administered chenodiol during pregnancy ( see Data ). The animal study findings have not been demonstrated with human use. The background risk of major birth defects and miscarriage for the indicated population is unknown. All pregnancies have a background risk of birth defect, loss, or other adverse outcomes. In the US general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2 to 4% and 15 to 20%, respectively. Data Animal Data Hepatic lesions were reported in neonatal baboons whose mothers had received 18 to 38 mg/kg of chenodiol throughout pregnancy (0.6 to 1.4 times the recommended human dose based on body surface area). Serious hepatic, renal and adrenal lesions were also reported in fetuses of female Rhesus monkeys given 60 to 90 mg/kg/day from GD 21-45 of pregnancy (1 to 2 times the recommended human dose based on body surface area). Non-human primates form sulfate conjugates of the known hepatotoxic bacterial metabolite of chenodiol, lithocholic acid, to a lesser extent than reported in humans, which may exaggerate the toxicity of orally dosed chenodiol compared to humans. However, there is also evidence that the hepatobiliary toxicity is partly due to the parent drug, chenodiol. 8.2 Lactation Risk Summary There are no data on the presence of chenodiol in either human or animal milk, the effects on the breastfed infant, or the effects on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for CTEXLI and any potential adverse effects on the breastfed infant from CTEXLI or the underlying maternal condition. 8.4 Pediatric Use The safety and effectiveness of CTEXLI for the treatment of CTX have not been established in pediatric patients. 8.5 Geriatric Use Trial 1 of CTEXLI in patients with CTX did not include patients aged 65 years and older."],"animal_pharmacology_and_or_toxicology":["13.2 Animal Toxicology and/or Pharmacology Chenodiol caused hepatobiliary toxicity (e.g., cholestasis) in many animal species, including rodents, non-rodents, and non-human primates at doses close to the human dose. Less efficient sulfation of the chenodiol metabolite, lithocholic acid, in non-human primates compared to humans is thought to cause the hepatobiliary toxicity of orally dosed chenodiol. However, there is evidence that the hepatobiliary toxicity is partly due to the parent drug, chenodiol."],"package_label_principal_display_panel":["PACKAGE/LABEL PRINCIPAL DISPLAY PANEL QR 2D; NDC: 79378-310-90; GTIN: XXXXXXXXXXXXXX; LOT: XXXXXX; EXP: YYYY-MM-DD; NDC 79378-310-90; Ctexli; (chenodiol); tablets; 250 mg; For oral use; 100 Tablets; Rx only; Recommended Dosage: see Prescribing Information. Store at 20°C to 25°C (68°F to 77°F), excursions permitted between 15°C to 30°C (59°F to 86°F) [see USP Controlled Room Temperature]. KEEP THIS AND ALL MEDICATION OUT OF THE REACH OF CHILDREN. Manufactured for Mirum Pharmaceuticals, Inc. Foster City, CA 94404 Ctexli 250 mg tablets bottle label"],"carcinogenesis_and_mutagenesis_and_impairment_of_fertility":["13.1 Carcinogenesis, Mutagenesis, Impairment of Fertility A two-year oral study of chenodiol in rats did not show a carcinogenic potential at the tested levels of 15 to 60 mg/kg/day (0.2 to 0.6 times the recommended human dose based on body surface area). In additional long-term studies, chenodiol given at oral doses up to 600 mg/kg/day in rats (6 times the recommended human dose based on body surface area), and 1000 mg/kg/day in mice (5 times the recommended human dose based on body surface area) induced benign and malignant liver cell tumors in female rats and cholangiomas in female rats and male mice. Two-year studies of lithocholic acid (a major metabolite of chenodiol) in mice (125 to 250 mg/kg/day, equivalent to 0.7 to 1.4 times the recommended human dose based on body surface area) and rats (250 and 500 mg/kg/day, equivalent to 3 to 5 times the recommended human dose based on body surface area) found it not to be carcinogenic. The dietary administration of lithocholic acid to chickens is reported to cause hepatic adenomatous hyperplasia."]},"tags":[{"label":"chenodiol","category":"class"},{"label":"Small Molecule","category":"modality"},{"label":"Bile acid receptor","category":"target"},{"label":"NR1H4","category":"gene"},{"label":"GPBAR1","category":"gene"},{"label":"FPR1","category":"gene"},{"label":"A05AA01","category":"atc"},{"label":"Oral","category":"route"},{"label":"Tablet","category":"form"},{"label":"Off-Patent","category":"patent"},{"label":"Generic Available","category":"availability"},{"label":"Mature","category":"status"},{"label":"Calculus in biliary tract","category":"indication"},{"label":"Approved 1980s","category":"decade"},{"label":"Cathartics","category":"pharmacology"},{"label":"Gastrointestinal Agents","category":"pharmacology"}],"phase":"marketed","safety":{"boxedWarnings":[],"safetySignals":[{"date":"","signal":"DIARRHOEA","source":"FDA FAERS","actionTaken":"41 reports"},{"date":"","signal":"HEPATIC ENZYME INCREASED","source":"FDA FAERS","actionTaken":"20 reports"},{"date":"","signal":"VOMITING","source":"FDA FAERS","actionTaken":"20 reports"},{"date":"","signal":"CONSTIPATION","source":"FDA FAERS","actionTaken":"19 reports"},{"date":"","signal":"DEATH","source":"FDA FAERS","actionTaken":"13 reports"},{"date":"","signal":"DRUG INEFFECTIVE","source":"FDA FAERS","actionTaken":"12 reports"},{"date":"","signal":"ABDOMINAL DISCOMFORT","source":"FDA FAERS","actionTaken":"9 reports"},{"date":"","signal":"DEHYDRATION","source":"FDA FAERS","actionTaken":"9 reports"},{"date":"","signal":"NAUSEA","source":"FDA FAERS","actionTaken":"9 reports"},{"date":"","signal":"HEADACHE","source":"FDA FAERS","actionTaken":"8 reports"}],"drugInteractions":[{"drug":"Bile acid sequestering agents (e.g., cholestyramine, colestipol)","severity":"major","mechanism":"Decreased absorption of CTEXLI in the intestine","management":"Avoid concomitant use of bile acid sequestering agents with CTEXLI","clinicalEffect":"Decreased efficacy of CTEXLI"},{"drug":"Aluminum-based antacids","severity":"major","mechanism":"Decreased absorption of CTEXLI in the intestine","management":"Avoid concomitant use of aluminum-based antacids with CTEXLI","clinicalEffect":"Decreased efficacy of CTEXLI"},{"drug":"Coumarin and its derivatives","severity":"major","mechanism":"CTEXLI may affect the pharmacodynamics of coumarin and its derivatives","management":"Monitor prothrombin time and adjust the dosage of coumarin or its derivatives accordingly","clinicalEffect":"Unexpected prolongation of the prothrombin time and hemorrhage"}],"commonSideEffects":[{"effect":"diarrhea","drugRate":"36%","_validated":true,"placeboRate":""},{"effect":"headache","drugRate":"21%","_validated":true,"placeboRate":""},{"effect":"abdominal pain (including abdominal pain upper)","drugRate":"14%","_validated":true,"placeboRate":""},{"effect":"constipation","drugRate":"14%","_validated":true,"placeboRate":""},{"effect":"hypertension","drugRate":"14%","_validated":true,"placeboRate":""},{"effect":"muscular weakness","drugRate":"14%","_validated":true,"placeboRate":""},{"effect":"upper respiratory tract infection","drugRate":"14%","_validated":true,"placeboRate":""}],"contraindications":["Diarrhea","Disease of liver","Gallstone Complications","Hypercholesterolemia","Liver function tests abnormal","Obstruction of bile duct","Pregnancy, function","Primary biliary cirrhosis","Sclerosing cholangitis"],"specialPopulations":{"Pregnancy":"Category X: See CONTRAINDICATIONS.","Paediatric use":"The safety and effectiveness of chenodiol in children have not been established."},"discontinuationRates":[{"trial":"Trial 1","drugArm":"7%","placeboArm":"","commonReason":"increased ALT levels > 3x ULN"}],"seriousAdverseEvents":[{"event":"increased ALT levels > 3x ULN","detail":"led to treatment interruption","severity":"serious","incidence":"7%"}]},"trials":[],"aliases":[],"patents":[],"pricing":[],"_sources":{"trials":{"url":"https://clinicaltrials.gov/search?intr=CHENODIOL","method":"api_direct","source":"ClinicalTrials.gov","rawText":"","confidence":1,"sourceType":"ctgov","retrievedAt":"2026-04-20T00:19:03.058733+00:00"},"regulatory.ca":{"url":"","method":"api_direct","source":"Health Canada DPD","rawText":"","confidence":1,"sourceType":"health_canada_dpd","retrievedAt":"2026-04-20T00:19:08.718258+00:00"},"regulatory.us":{"url":"","method":"api_direct","source":"FDA Drugs@FDA","rawText":"","confidence":1,"sourceType":"fda_drugsfda","retrievedAt":"2026-04-20T00:19:02.188820+00:00"},"publicationCount":{"url":"https://pubmed.ncbi.nlm.nih.gov/?term=CHENODIOL","method":"api_direct","source":"PubMed/NCBI","rawText":"","confidence":1,"sourceType":"pubmed","retrievedAt":"2026-04-20T00:19:09.028484+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":"2026-04-20T00:19:01.649960+00:00"},"administration.route":{"url":"","method":"deterministic","source":"FDA Label","rawText":"","confidence":1,"sourceType":"fda_label","retrievedAt":"2026-04-20T00:19:01.649985+00:00"},"safety.boxedWarnings":{"url":"","method":"deterministic","source":"FDA Label (no boxed warning)","rawText":"","confidence":1,"sourceType":"fda_label","retrievedAt":"2026-04-20T00:19:01.649989+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":"2026-04-20T00:19:10.479307+00:00"},"mechanism.target_chembl":{"url":"","method":"api_direct","source":"ChEMBL mechanism: Bile acid receptor FXR agonist","rawText":"","confidence":1,"sourceType":"chembl","retrievedAt":"2026-04-20T00:19:10.085809+00:00"},"safety.drugInteractions":{"url":"","method":"ai_extraction","source":"FDA Label + AI","aiModel":"featherless","rawText":"","confidence":0.9,"sourceType":"fda_label","retrievedAt":"2026-04-20T00:19:45.581626+00:00"},"crossReferences.chemblId":{"url":"https://www.ebi.ac.uk/chembl/compound_report_card/CHEMBL240597/","method":"api_direct","source":"ChEMBL (EMBL-EBI)","rawText":"","confidence":1,"sourceType":"chembl","retrievedAt":"2026-04-20T00:19:09.739767+00:00"},"safety.commonSideEffects":{"url":"","method":"ai_extraction","source":"FDA Label + AI","aiModel":"featherless","rawText":"6 ADVERSE REACTIONS The following clinically significant adverse reaction is described elsewhere in the labeling: • Hepatotoxicity [see Warnings and Precautions ( 5.1 )] The most common adverse reactions (incidence > 14%) are diarrhea, headache, abdominal pain, constipation, hypertension, muscular weakness, and upper respiratory tract infection. ( 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 Cli","confidence":0.95,"sourceType":"fda_label","retrievedAt":"2026-04-20T00:19:29.716592+00:00"},"regulatory.fda_application":{"url":"","method":"deterministic","source":"FDA Label","rawText":"NDA219488","confidence":1,"sourceType":"fda_label","retrievedAt":"2026-04-20T00:19:01.649992+00:00"}},"allNames":"chenix","offLabel":[],"synonyms":["chenodiol","chenodeoxycholic acid","chenic acid","anthropodesoxycholic acid","anthropododesoxycholic acid","chenodesoxycholic acid","gallodesoxycholic acid"],"timeline":[{"date":"1983-07-28","type":"positive","source":"DrugCentral","milestone":"FDA approval"},{"date":"2009-10-22","type":"neutral","source":"FDA Orange Book","milestone":"Generic entry — 1 manufacturer approved"},{"date":"2028-02-21","type":"negative","source":"FDA Orange Book","milestone":"New Product exclusivity expires"}],"aiSummary":"Chenix (chenodiol) is a small molecule drug that targets the bile acid receptor. Originally developed and currently owned by Mirum, it was FDA-approved in 1983 for the treatment of calculus in the biliary tract. Chenix is an off-patent, generic medication with a half-life of 1.42 hours. It is used to dissolve gallstones by altering the composition of bile acids. As an off-patent medication, it is available from a single generic manufacturer.","approvals":[{"date":"1983-07-28","orphan":false,"company":"","regulator":"FDA"}],"brandName":"Chenix","ecosystem":[{"indication":"Calculus in biliary tract","otherDrugs":[{"name":"cholic acid","slug":"cholic-acid","company":"Rtrx"},{"name":"ursodiol","slug":"ursodiol","company":"Actavis Labs Ut Inc"}],"globalPrevalence":null}],"mechanism":{"target":"Bile acid receptor","novelty":"Follow-on","targets":[{"gene":"NR1H4","source":"DrugCentral","target":"Bile acid receptor","protein":"Bile acid receptor"},{"gene":"GPBAR1","source":"DrugCentral","target":"G-protein coupled bile acid receptor 1","protein":"G-protein coupled bile acid receptor 1"},{"gene":"FPR1","source":"DrugCentral","target":"fMet-Leu-Phe receptor","protein":"fMet-Leu-Phe receptor"}],"modality":"Small Molecule","drugClass":"Bile Acid [EPC]","explanation":"Imagine your body's bile acids as a sticky liquid that helps digest food. Chenix helps change the way your body uses this liquid, making it less sticky and more likely to dissolve gallstones. This process helps to break down and remove gallstones from the biliary tract.","oneSentence":"Chenix works by binding to the bile acid receptor, which increases the excretion of bile acids and helps dissolve gallstones.","technicalDetail":"Chenix acts as a bile acid receptor agonist, increasing the expression of fibroblast growth factor 19 (FGF19), which in turn inhibits the expression of cholesterol 7-alpha-hydroxylase (CYP7A1), leading to a decrease in bile acid synthesis and an increase in bile acid excretion."},"commercial":{"launchDate":"1983","_launchSource":"DrugCentral (FDA 1983-07-28, )"},"references":[{"id":1,"url":"https://drugcentral.org/drugcard/4361","fields":["approvals","synonyms","ATC","PK","indications","contraindications","DDIs","targets","patents","FAERS"],"source":"DrugCentral"},{"id":2,"url":"https://clinicaltrials.gov/search?intr=CHENODIOL","fields":["trials"],"source":"ClinicalTrials.gov"},{"id":3,"url":"https://pubmed.ncbi.nlm.nih.gov/?term=CHENODIOL","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"}],"_enrichedAt":"2026-03-30T09:31:57.475695","_validation":{"fieldsValidated":1,"lastValidatedAt":"2026-04-20T00:19:46.125220+00:00","fieldsConflicting":0,"overallConfidence":0.95},"biosimilars":[],"competitors":[{"drugName":"ursodiol","drugSlug":"ursodiol","fdaApproval":"1987-12-31","genericCount":19,"patentStatus":"Off-patent — generic available","relationship":"same-class"},{"drugName":"cholic acid","drugSlug":"cholic-acid","fdaApproval":"2015-03-17","patentStatus":"Unknown","relationship":"same-class"},{"drugName":"obeticholic acid","drugSlug":"obeticholic-acid","fdaApproval":"2016-05-27","patentExpiry":"Sep 6, 2033","patentStatus":"Patent protected","relationship":"same-class"}],"exclusivity":[{"code":"NP","date":"Feb 21, 2028"}],"genericName":"chenodiol","indications":{"approved":[{"name":"Calculus in biliary tract","source":"DrugCentral","snomedId":266474003,"regulator":"FDA","eligibility":"patients with radiolucent stones in well-opacifying gallbladders, in whom selective surgery would be undertaken except for the presence of increased surgical risk due to systemic disease or age"}],"offLabel":[],"pipeline":[]},"currentOwner":"Mirum","drugCategory":"mature","labelChanges":[],"patentStatus":"Off-patent — no active Orange Book patents","relatedDrugs":[{"drugId":"ursodiol","brandName":"ursodiol","genericName":"ursodiol","approvalYear":"1987","relationship":"same-class"},{"drugId":"cholic-acid","brandName":"cholic acid","genericName":"cholic acid","approvalYear":"2015","relationship":"same-class"},{"drugId":"obeticholic-acid","brandName":"obeticholic acid","genericName":"obeticholic acid","approvalYear":"2016","relationship":"same-class"}],"trialDetails":[{"nctId":"NCT07005752","phase":"PHASE4","title":"A Study to Evaluate the Efficacy and Safety of CnU Capsule 750 mg in Patients With Cholesterol Gallstones(GB Stones).","status":"RECRUITING","sponsor":"Myungmoon Pharma. Co. Ltd.","startDate":"2025-07-28","conditions":["Cholesterol Cholelithiasis"],"enrollment":484,"completionDate":"2026-06"},{"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":"NCT03406780","phase":"PHASE2","title":"A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy","status":"COMPLETED","sponsor":"Capricor Inc.","startDate":"2018-04-04","conditions":["Muscular Dystrophies","Muscular Dystrophy, Duchenne","Muscular Disorders, Atrophic","Muscular Diseases","Neuromuscular Diseases","Nervous System Diseases","Genetic Diseases, X-Linked","Genetic Diseases, Inborn"],"enrollment":20,"completionDate":"2020-03-10"},{"nctId":"NCT04623671","phase":"PHASE2","title":"Intravenous Infusion of CAP-1002 in Patients With COVID-19","status":"COMPLETED","sponsor":"Capricor Inc.","startDate":"2020-11-15","conditions":["Covid19"],"enrollment":55,"completionDate":"2022-02-04"},{"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":"NCT04270682","phase":"PHASE3","title":"Study to Evaluate Patients With Cerebrotendinous Xanthomatosis (RESTORE)","status":"COMPLETED","sponsor":"Mirum Pharmaceuticals, Inc.","startDate":"2020-01-31","conditions":["CTX"],"enrollment":19,"completionDate":"2023-10-04"},{"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":"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":"NCT01585025","phase":"PHASE2","title":"Obeticholic Acid in Bile Acid Diarrhoea","status":"COMPLETED","sponsor":"Imperial College Healthcare NHS Trust","startDate":"2012-04","conditions":["Primary Bile Acid Malabsorption","Secondary Bile Acid Malabsorption","Chronic Diarrhoea"],"enrollment":35,"completionDate":"2014-02"},{"nctId":"NCT01865812","phase":"PHASE2","title":"Phase 2 Study on Effects of Obeticholic Acid (OCA) on Lipoprotein Metabolism in Participants With Primary Biliary Cirrhosis","status":"COMPLETED","sponsor":"Intercept Pharmaceuticals","startDate":"2013-12-03","conditions":["Primary Biliary Cirrhosis"],"enrollment":27,"completionDate":"2016-09-12"},{"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":"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":"NCT00570765","phase":"PHASE2","title":"Study of INT-747 as Monotherapy in Participants With Primary Biliary Cirrhosis (PBC)","status":"COMPLETED","sponsor":"Intercept Pharmaceuticals","startDate":"2008-01-17","conditions":["Liver Cirrhosis, Biliary"],"enrollment":60,"completionDate":"2017-09-25"},{"nctId":"NCT01473524","phase":"PHASE3","title":"Phase 3 Study of Obeticholic Acid in Patients With Primary Biliary Cirrhosis","status":"COMPLETED","sponsor":"Intercept Pharmaceuticals","startDate":"2012-01","conditions":["Primary Biliary Cirrhosis"],"enrollment":217,"completionDate":"2018-12-17"},{"nctId":"NCT02876484","phase":"PHASE4","title":"Effects of Bile Acids and Bile Acid Sequestrants on GLP-1 Secretion After Roux-en-Y Gastric Bypass","status":"COMPLETED","sponsor":"Hvidovre University Hospital","startDate":"2016-06","conditions":["Severe Obesity"],"enrollment":12,"completionDate":"2020-11"},{"nctId":"NCT02952963","phase":"PHASE4","title":"Effect of Bile Acids and Bile Acid Sequstrants on GLP-1 Secretion After Roux-en-Y Gastric Bypass","status":"COMPLETED","sponsor":"Hvidovre University Hospital","startDate":"2016-10","conditions":["Severe Obesity"],"enrollment":8,"completionDate":"2020-11"},{"nctId":"NCT03059537","phase":"PHASE4","title":"Validation of Stimulated ∆FGF19 for Diagnosing Bile Acid Diarrhoea","status":"COMPLETED","sponsor":"Lars Kristian Munck","startDate":"2017-03-13","conditions":["Bile Acid Malabsorption"],"enrollment":71,"completionDate":"2017-11-27"},{"nctId":"NCT02979210","phase":"NA","title":"Infant Fecal Insult Study","status":"COMPLETED","sponsor":"Kimberly-Clark Corporation","startDate":"2016-11","conditions":["Dermatitis"],"enrollment":24,"completionDate":"2017-02-01"},{"nctId":"NCT02340247","phase":"PHASE4","title":"Effects of Bile Acids on GLP-1 Secretion After Roux-en-Y Gastric Bypass","status":"COMPLETED","sponsor":"Hvidovre University Hospital","startDate":"2014-11","conditions":["Severe Obesity"],"enrollment":11,"completionDate":""},{"nctId":"NCT00004442","phase":"NA","title":"Study of Bile Acids in Patients With Peroxisomal Disorders","status":"TERMINATED","sponsor":"University of Cincinnati","startDate":"","conditions":["Infantile Refsum's Disease","Zellweger Syndrome","Bifunctional Enzyme Deficiency","Adrenoleukodystrophy"],"enrollment":25,"completionDate":"1999-04"},{"nctId":"NCT01914562","phase":"PHASE1","title":"Effect of Food on Pharmacokinetics of Obeticholic Acid (OCA)","status":"COMPLETED","sponsor":"Intercept Pharmaceuticals","startDate":"2013-08","conditions":["Healthy"],"enrollment":32,"completionDate":"2013-11"},{"nctId":"NCT01666223","phase":"NA","title":"Effect of Bile Acids on GLP-1 Secretion","status":"COMPLETED","sponsor":"University Hospital, Gentofte, Copenhagen","startDate":"2012-11","conditions":["Type 2 Diabetes","Obesity"],"enrollment":20,"completionDate":"2013-06"},{"nctId":"NCT01933503","phase":"PHASE1","title":"Single Dose and Multiple Dose Trial to Assess Pharmacokinetics of Obeticholic Acid (OCA)","status":"COMPLETED","sponsor":"Intercept Pharmaceuticals","startDate":"2013-10","conditions":["Healthy"],"enrollment":24,"completionDate":"2013-11"},{"nctId":"NCT01904539","phase":"PHASE1","title":"Hepatic Impairment Trial of Obeticholic Acid","status":"COMPLETED","sponsor":"Intercept Pharmaceuticals","startDate":"2013-06","conditions":["Hepatic Impairment"],"enrollment":32,"completionDate":"2013-10"},{"nctId":"NCT00018694","phase":"NA","title":"Cholestanol in Humans","status":"WITHDRAWN","sponsor":"US Department of Veterans Affairs","startDate":"1999-10","conditions":["Cerebrotendinous Xanthomatosis"],"enrollment":0,"completionDate":"2009-12"},{"nctId":"NCT01674946","phase":"PHASE1","title":"Effect of Bile Acids on the Secretion of Satiation Peptides in Humans","status":"COMPLETED","sponsor":"University Hospital, Basel, Switzerland","startDate":"2011-09","conditions":["Echolocation"],"enrollment":12,"completionDate":"2012-05"},{"nctId":"NCT00912301","phase":"PHASE2","title":"Cheno Effect on Transit in Health and IBS-C","status":"COMPLETED","sponsor":"Mayo Clinic","startDate":"2009-04","conditions":["Constipation-predominant Irritable Bowel Syndrome"],"enrollment":36,"completionDate":"2009-12"},{"nctId":"NCT00465751","phase":"EARLY_PHASE1","title":"Effects of FXR Activation on Hepatic Lipid and Glucose Metabolism","status":"COMPLETED","sponsor":"University Hospital, Basel, Switzerland","startDate":"2004-10","conditions":["Metabolic Syndrome","Familial Hypertriglyceridemia","Familial Combined Hyperlipidemia"],"enrollment":30,"completionDate":""},{"nctId":"NCT00004346","phase":"PHASE2","title":"Phase II Study of Cholesterol- and Cholestanol-Free Diet, Lovastatin, and Chenodeoxycholic Acid for Cerebrotendinous Xanthomatosis","status":"UNKNOWN","sponsor":"National Center for Research Resources (NCRR)","startDate":"1996-01","conditions":["Cerebrotendinous Xanthomatosis"],"enrollment":5,"completionDate":""}],"genericFilers":[],"latestUpdates":[],"manufacturing":[],"administration":{"route":"Oral","formulation":"Tablet","formulations":[{"form":"TABLET, FILM COATED","route":"ORAL","productName":"Chenodal"},{"form":"TABLET, FILM COATED","route":"ORAL","productName":"Chenodiol"}]},"_patentsChecked":true,"crossReferences":{"NUI":"N0000146754","MMSL":"25632","NDDF":"001208","UNII":"0GEI24LG0J","VUID":"4022365","CHEBI":"CHEBI:16755","VANDF":"4018428","INN_ID":"4391","RXNORM":"2323","UMLSCUI":"C0008024","chemblId":"CHEMBL240597","ChEMBL_ID":"CHEMBL240597","KEGG_DRUG":"D00163","DRUGBANK_ID":"DB06777","PDB_CHEM_ID":" JN3","PUBCHEM_CID":"10133","SNOMEDCT_US":"82490009","IUPHAR_LIGAND_ID":"608","MESH_DESCRIPTOR_UI":"D002635"},"formularyStatus":[],"_enricherVersion":"v2","developmentCodes":[],"ownershipHistory":[{"period":"present","companyName":"Mirum","relationship":"Current Owner"}],"pharmacokinetics":{"source":"DrugCentral","halfLife":"1.42 hours","clearance":"5.7 mL/min/kg","volumeOfDistribution":"0.19 L/kg"},"publicationCount":51,"therapeuticAreas":["Metabolic"],"atcClassification":{"source":"DrugCentral","atcCode":"A05AA01","allCodes":["A05AA01"]},"biosimilarFilings":[],"recentPublications":[{"date":"2025 Dec","pmid":"41324091","title":"FDA Approves First Targeted Treatment for Cerebrotendinous Xanthomatosis: A Perspective on a Landmark in Rare Lipid Storage Disease Therapy.","journal":"Health science reports"},{"date":"2006","pmid":"40864842","title":"Chenodiol.","journal":""},{"date":"2025 Jul","pmid":"40443237","title":"Regulatory News: Chenodiol for the Treatment of Cerebrotendinous Xanthomatosis: FDA Approval Summary.","journal":"Journal of inherited metabolic disease"},{"date":"2025 Jun","pmid":"40185658","title":"CTEXLI (chenodiol).","journal":"Clinical therapeutics"},{"date":"2022 Oct","pmid":"35899436","title":"Differential effects of metformin-mediated BSEP repression on pravastatin and bile acid pharmacokinetics in humans: A randomized controlled trial.","journal":"Clinical and translational science"}],"companionDiagnostics":[],"genericManufacturers":1,"_genericFilersChecked":true,"genericManufacturerList":["Lgm Pharma"],"status":"approved","companyName":"Mirum","companyId":"","modality":"Small molecule","firstApprovalDate":"1983","enrichmentLevel":4,"visitCount":1,"regulatoryByCountry":[{"country_code":"US","regulator":"FDA","status":"approved","approval_date":"1983-07-28T00:00:00.000Z","mah":"","brand_name_local":null,"application_number":""},{"country_code":"US","regulator":"FDA","status":"approved","approval_date":"2009-10-22T00:00:00.000Z","mah":"LGM PHARMA","brand_name_local":null,"application_number":"ANDA091019"},{"country_code":"US","regulator":"FDA","status":"approved","approval_date":"2025-02-21T00:00:00.000Z","mah":"MIRUM","brand_name_local":null,"application_number":"NDA219488"},{"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},{"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":"IL","regulator":"MOH","status":"likely_approved","approval_date":null,"mah":null,"brand_name_local":null,"application_number":null},{"country_code":"IL","regulator":"MOH","status":"likely_approved","approval_date":null,"mah":null,"brand_name_local":null,"application_number":null},{"country_code":"IL","regulator":"MOH","status":"likely_approved","approval_date":null,"mah":null,"brand_name_local":null,"application_number":null},{"country_code":"SG","regulator":"HSA","status":"likely_approved","approval_date":null,"mah":null,"brand_name_local":null,"application_number":null},{"country_code":"SG","regulator":"HSA","status":"likely_approved","approval_date":null,"mah":null,"brand_name_local":null,"application_number":null},{"country_code":"SG","regulator":"HSA","status":"likely_approved","approval_date":null,"mah":null,"brand_name_local":null,"application_number":null},{"country_code":"AE","regulator":"MOH","status":"likely_approved","approval_date":null,"mah":null,"brand_name_local":null,"application_number":null},{"country_code":"AE","regulator":"MOH","status":"likely_approved","approval_date":null,"mah":null,"brand_name_local":null,"application_number":null},{"country_code":"AE","regulator":"MOH","status":"likely_approved","approval_date":null,"mah":null,"brand_name_local":null,"application_number":null},{"country_code":"KR","regulator":"MFDS","status":"likely_approved","approval_date":null,"mah":null,"brand_name_local":null,"application_number":null},{"country_code":"KR","regulator":"MFDS","status":"likely_approved","approval_date":null,"mah":null,"brand_name_local":null,"application_number":null},{"country_code":"KR","regulator":"MFDS","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":"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":"GB","regulator":"MHRA","status":"likely_approved","approval_date":null,"mah":null,"brand_name_local":null,"application_number":null},{"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}],"trialStats":{"total":0,"withResults":0},"validation":{"fieldsValidated":1,"lastValidatedAt":"2026-04-20T00:19:46.125220+00:00","fieldsConflicting":0,"overallConfidence":0.95},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}