{"id":"imiglucerase","rwe":[{"pmid":"41821052","year":"2026","title":"The global impact of imiglucerase therapy in children with Gaucher disease types 1 and 3: a real-world analysis from the International Collaborative Gaucher Group Gaucher Registry.","finding":"","journal":"Orphanet journal of rare diseases","studyType":"Clinical Study"},{"pmid":"41714274","year":"2026","title":"Maternal and Fetal Outcomes in Imiglucerase-Treated Patients With Gaucher Disease: Real-World Evidence From the International Collaborative Gaucher Group (ICGG) Gaucher Registry Pregnancy Sub-Registry.","finding":"","journal":"Journal of inherited metabolic disease","studyType":"Clinical Study"},{"pmid":"30000979","year":"2006","title":"Taliglucerase alfa.","finding":"","journal":"","studyType":"Clinical Study"},{"pmid":"41645321","year":"2026","title":"Investigating the therapeutic profile of velaglucerase alfa in paediatric patients with Gaucher disease: a systematic review across all paediatric age groups.","finding":"","journal":"Orphanet journal of rare diseases","studyType":"Clinical Study"},{"pmid":"41599285","year":"2026","title":"Quantitative and Comparative Assessment of Recombinant Human β-Glucocerebrosidase Uptake Bioactivity Using a Stable hMMR-Expressing CHO Cell Model.","finding":"","journal":"Molecules (Basel, Switzerland)","studyType":"Clinical Study"}],"_fda":{"id":"6a32f852-ba91-4185-a618-0ba55a14442e","set_id":"df60f030-866b-4374-a31f-8ae3f6b45c38","openfda":{"nui":["M0009360","N0000175820"],"unii":["Q6U6J48BWY"],"route":["INTRAVENOUS"],"rxcui":["1726266","1726268"],"spl_id":["6a32f852-ba91-4185-a618-0ba55a14442e"],"brand_name":["Cerezyme"],"spl_set_id":["df60f030-866b-4374-a31f-8ae3f6b45c38"],"package_ndc":["58468-4663-1"],"product_ndc":["58468-4663"],"generic_name":["IMIGLUCERASE"],"product_type":["HUMAN PRESCRIPTION DRUG"],"pharm_class_cs":["Glucosylceramidase [CS]"],"substance_name":["IMIGLUCERASE"],"pharm_class_epc":["Hydrolytic Lysosomal Glucocerebroside-specific Enzyme [EPC]"],"manufacturer_name":["Genzyme Corporation"],"application_number":["BLA020367"],"is_original_packager":[true]},"version":"26","pregnancy":["8.1 Pregnancy Pregnancy Exposure Registry There is a pregnancy exposure registry that monitors pregnancy outcomes in women exposed to CEREZYME during pregnancy. Pregnant women exposed to CEREZYME and health care providers are encouraged to contact the Gaucher patient registry at 1-800-745-4447, extension 15500 or visit www.registrynxt.com. Risk Summary Available data on more than 500 pregnancies from the international Gaucher Disease registry, postmarketing reports, published observational studies and case reports with CEREZYME or non–US-licensed imiglucerase use in pregnant women have not identified a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes. There are risks associated with symptomatic Type I Gaucher disease in pregnancy (see Clinical Considerations ) . No animal reproduction studies have been conducted with imiglucerase. The estimated background risk of major birth defects and miscarriage in the indicated population is unknown. All pregnancies have a background risk of birth defect, loss, or other adverse outcomes. In the U.S. 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. Clinical Considerations Disease-Associated Maternal and/or Embryo/Fetal Risk Pregnancy may exacerbate existing Type 1 Gaucher disease symptoms or result in new disease manifestations. Untreated symptomatic Type 1 Gaucher disease may lead to complications during pregnancy, including hepatosplenomegaly, which can interfere with the normal growth of a pregnancy and thrombocytopenia, which can lead to excessive bleeding."],"description":["11 DESCRIPTION Imiglucerase is a hydrolytic lysosomal glucocerebrosidase-specific enzyme. It is an analogue of the human enzyme b-glucocerebrosidase (b-D-glucosyl-N-acylsphingosine glucohydrolase, E.C. 3.2.1.45), produced by recombinant DNA technology using mammalian cell culture (Chinese hamster ovary). Purified imiglucerase is a monomeric glycoprotein of 497 amino acids, containing 4 N-linked glycosylation sites (Mr=60,430). Imiglucerase differs from placental glucocerebrosidase by one amino acid at position 495, where histidine is substituted for arginine. The oligosaccharide chains at the glycosylation sites have been modified to terminate in mannose sugars. The modified carbohydrate structures on imiglucerase are somewhat different from those on placental glucocerebrosidase. CEREZYME (imiglucerase) for injection is intended for intravenous use. It is supplied as a sterile, nonpyrogenic, white to off-white lyophilized powder for reconstitution with Sterile Water for Injection, USP. Each single-dose vial contains 424 units imiglucerase, mannitol (340 mg), polysorbate 80, NF (1.06 mg), and sodium citrates: disodium hydrogen citrate (36 mg) and trisodium citrate (104 mg). An enzyme unit (U) is defined as the amount of enzyme that catalyzes the hydrolysis of 1 micromole of the synthetic substrate para-nitrophenyl-b-D-glucopyranoside (pNP-Glc) per minute at 37°C. Reconstituted solutions have a pH of approximately 6.1."],"how_supplied":["16 HOW SUPPLIED/STORAGE AND HANDLING CEREZYME (imiglucerase) for injection is supplied as a white to off-white lyophilized powder in a carton containing one single-dose vial: NDC 58468-4663-1. Each vial contains 400 units of imiglucerase. CEREZYME does not contain any preservatives. Store refrigerated at 2°C to 8°C (36°F to 46°F). For storage of reconstituted and diluted solution [see Dosage and Administration (2.2) ] ."],"boxed_warning":["WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. Initiate CEREZYME in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue CEREZYME and immediately initiate appropriate medical treatment, including use of epinephrine. Inform patients of the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis and to seek immediate medical care should symptoms occur [see Warnings and Precautions (5.1) ] . WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS See full prescribing information for complete boxed warning . Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. ( 5.1 ) Initiate CEREZYME in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. ( 5.1 ) If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue CEREZYME and immediately initiate appropriate medical treatment, including use of epinephrine. ( 5.1 )"],"geriatric_use":["8.5 Geriatric Use Among the patients with Type 1 Gaucher disease in Study 3 [see Clinical Studies (14) ] who had hemoglobin or platelet count measurements 1 to 3 years after initiating CEREZYME (n = 1,053), 6% of patients were 65 and over and 1% were 75 and over. No overall differences in effectiveness of CEREZYME were observed between patients 65 years of age and older and younger adult patients with Type 1 Gaucher disease. The safety evaluation for patients 65 years of age and older with Type 1 Gaucher disease was conducted using data from a pharmacovigilance database that collected individual case safety reports and did not reveal new safety findings. Clinical studies of CEREZYME did not include sufficient numbers of patients 65 years of age and older to detect differences in safety between older and younger adult patients with Type 1 Gaucher disease. Clinical studies of CEREZYME did not include sufficient numbers of patients 65 years of age and older to detect differences in efficacy and safety between older and younger adult patients with Type 3 Gaucher disease."],"pediatric_use":["8.4 Pediatric Use The safety and effectiveness of CEREZYME for the treatment of non-CNS manifestations of Type 1 or Type 3 Gaucher disease have been established in pediatric patients. Use of CEREZYME for the treatment of non-CNS manifestations of Type 1 Gaucher disease is supported by evidence from adequate and well-controlled trials in adults and pediatric patients 12 years of age and older and additional safety and efficacy data from an observational study of Type 1 Gaucher disease in pediatric patients [see Adverse Reactions (6.1) and Clinical Studies (14) ] . Use of CEREZYME for the treatment of non-CNS manifestations of Type 3 Gaucher disease is supported by evidence from adequate and well-controlled trials in adults and pediatric patients 12 years of age and older with Type I Gaucher disease and additional safety and efficacy data from an observational study of Type 3 Gaucher disease in adults and pediatric patients [see Adverse Reactions (6.1) and Clinical Studies (14) ] ."],"effective_time":"20251222","clinical_studies":["14 CLINICAL STUDIES Study 1 was a randomized, double-blind, active-controlled clinical trial that enrolled 30 patients (17 male and 13 female) with Type 1 Gaucher disease. Patient ages ranged from 12 to 69 years, with a mean age of 38 years in the CEREZYME treatment group and a mean age of 28 years in the alglucerase treatment group at baseline. The inclusion criteria required patients to have a hemoglobin level of at least 1 g/dL below the lower limit of the normal range for their respective age and sex. Patients were randomized 1:1 to receive either CEREZYME 60 units/kg administered intravenously every other week or alglucerase for 6 months. In Study 1, the primary efficacy parameters were an increase in hemoglobin concentration (at least 1 g/dL) and platelet count and a decrease in spleen and liver volume at 6 months. Efficacy results are shown in Table 3. In Study 1, bone x-rays showed improvements in cortical thickness and lucencies in 7 of 11 CEREZYME-treated patients. Table 3: Change from Baseline to Month 6 in Clinical Efficacy Parameters in a Randomized, Double-Blind Active-Controlled Trial of CEREZYME Compared to Alglucerase in Patients 12 Years of Age and Older with Gaucher Disease Type 1 (Study 1) Clinical Parameter CEREZYME (N=15) Alglucerase (N=15) Difference (CEREZYME – Alglucerase) [95% CI] Confidence intervals were calculated using the t distribution (appropriate for small sample sizes) and the standard error of the difference in sample means (i.e., the pooled estimate of the common standard deviation, computed as the weighted average of the standard deviations in the two treatment groups); there was no evidence that the assumption of equal variances between the groups was violated. Hemoglobin concentration (g/dL) Baseline 10.7 10.9 – Absolute Change from Baseline 1.9 1.6 0.3 [-0.6, 1.3] Platelet count (× 10 3 /mm 3 ) Baseline 68.5 74.2 – Absolute Change from Baseline 22.7 15.8 6.9 [-10.4, 24.1] Liver volume (mL) Baseline 2521 2788 – Absolute Change from Baseline -310 -307 -3 [-246, 240] Percent Change from Baseline (%) -11 -10 -1 [-9, 7] Spleen volume (mL) Baseline 2369 2603 – Absolute Change from Baseline -902 -874 -28 [-652, 596] Percent Change from Baseline (%) -35 -30 -5 [-14, 4] In Study 2, an open-label extension study of Study 1, 29 patients with Type 1 Gaucher disease continued their assigned treatment for an additional 18 months. Patients were unblinded 3 months into Study 2 and those on alglucerase were allowed to cross-over to CEREZYME treatment. After total treatment duration of CEREZYME for 18–24 months, mean increase from baseline of Study 1 in hemoglobin was 2.4 g/dL, mean increase in platelet count was 40 ×10 3 /mm 3 , mean change in liver volume was -20%, and mean change in spleen volume was -57%. The efficacy of CEREZYME for the treatment of non-CNS manifestations of Type 1 and Type 3 Gaucher disease was assessed in Study 3, an observational study, using data from the International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry (NCT00358943). Study 3 included patients with Type 1 or Type 3 Gaucher disease who were treated with CEREZYME as initial therapy with an index clinical assessment and one or more follow-up clinical assessments. Study 3 was a baseline-controlled analysis in patients with Type 1 Gaucher disease (19 weeks to 87 years of age) and patients with Type 3 Gaucher disease (7 weeks to 54 years of age) who received CEREZYME intravenously as prescribed by their physicians (initiated treatment between 1992–2021). After approximately two years (1 to 3 years) of CEREZYME treatment in patients with Type 1 and 3 Gaucher disease, mean changes from baseline in the following measures showed improvement: hemoglobin, platelet count, liver volume, spleen volume, and height Z-score. Among 1,052 Type 1 Gaucher disease patients, mean baseline hemoglobin was 11.8 g/dL and mean increase from baseline was 1.5 g/dL (95% CI: 1.4, 1.5). Among 1,053 Type 1 Gaucher disease patients, mean baseline platelet count was 128×10 3 /mm 3 and mean increase from baseline was 64×10 3 /mm 3 (95% CI: 59.6, 67.9). Among 118 Type 3 Gaucher disease patients, mean baseline hemoglobin levels were 10 g/dL and mean increase from baseline was 1.8 g/dL (95% CI: 1.5, 2.1). Among 116 Type 3 Gaucher disease patients, mean baseline platelet count was 149×10 3 /mm 3 and mean increase from baseline was 105×10 3 /mm 3 (95% CI: 87.4, 122.4). The 2-year summaries include measurements within 1 to 3 years after treatment initiation due to the lack of predefined data collection timepoints in the registry."],"pharmacodynamics":["12.2 Pharmacodynamics No formal pharmacodynamic studies have been conducted with CEREZYME."],"pharmacokinetics":["12.3 Pharmacokinetics During one-hour intravenous infusions of four doses (7.5, 15, 30, 60 units/kg) of CEREZYME, steady-state enzymatic activity was achieved by 30 minutes. Following infusion, the half-life of plasma enzymatic activity ranged from 3.6 to 10.4 minutes. Plasma clearance ranged from 9.8 to 20.3 mL/min/kg (mean ± SD, 14.5 ± 4.0 mL/min/kg). The volume of distribution corrected for weight ranged from 0.09 to 0.15 L/kg (mean ± SD, 0.12 ± 0.02 L/kg). These variables do not appear to be influenced by dose or duration of infusion. However, only one or two patients were studied at each dose level and infusion rate. Antidrug Antibody Effects on Pharmacokinetics In patients who developed IgG antibody to CEREZYME, an apparent effect on serum enzyme levels resulted in diminished volume of distribution and clearance and increased elimination half-life compared to patients without antibody [see Adverse Reactions (6.2) ] ."],"adverse_reactions":["6 ADVERSE REACTIONS The following clinically significant adverse reactions are described elsewhere in the labeling: Hypersensitivity Reactions Including Anaphylaxis [see Warnings and Precautions (5.1) ] Infusion-Associated Reactions [see Warnings and Precautions (5.2) ] Adverse reactions reported in adults and pediatric patients include back pain, chills, dizziness, fatigue, headache, hypersensitivity reactions, nausea, pyrexia, and vomiting. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Genzyme at 1-800-633-1610 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials and Postmarketing Experience The following adverse reactions associated with the use of imiglucerase were identified in clinical studies or postmarketing reports. Because some of these reactions were 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. See Table 2 for adverse reactions occurring in adults and pediatric patients treated with CEREZYME in clinical trials and the postmarketing setting. Table 2: Adverse Reactions in Adults and Pediatric Patients Treated with CEREZYME Adverse Reactions Nervous system disorders dizziness, headache Cardiac disorders tachycardia Vascular disorders cyanosis, Signs and symptoms suggestive of hypersensitivity reactions including anaphylaxis [see Warnings and Precautions (5.1) ] and other infusion-associated reactions [see Warnings and Precautions (5.2) ] . flushing, hypotension, hypertension Respiratory, thoracic and mediastinal disorders cough, dyspnea, pneumonia, pulmonary hypertension Gastrointestinal disorders abdominal pain, diarrhea, nausea, vomiting Immune system disorders anaphylaxis, hypersensitivity Skin and subcutaneous tissue disorders angioedema, pruritus, rash, urticaria Musculoskeletal and connective tissue disorders back pain General disorders and administration site conditions chest discomfort, chills, fatigue, infusion-site burning, infusion-site discomfort, infusion-site swelling, pyrexia 6.2 Immunogenicity The observed incidence of anti-drug antibodies (ADA) is highly dependent on the sensitivity and specificity of the assay. Differences in assay methods preclude meaningful comparisons of the incidence of ADA in the studies described below with the incidence of ADA in other studies, including those of CEREZYME or of other imiglucerase products. Approximately 15% of patients treated and tested to date have developed IgG antibody to CEREZYME during the first year of therapy. Patients who developed IgG antibody did so largely within 6 months of treatment and rarely developed antibodies to CEREZYME after 12 months of therapy. Approximately 46% of patients with detectable IgG antibodies experienced symptoms of hypersensitivity. Patients with antibody to CEREZYME have higher risk of hypersensitivity reaction [see Warnings and Precautions (5.1) ] . Patients who developed IgG antibody to CEREZYME had increased elimination half-life compared to patients without antibody [see Clinical Pharmacology (12.3) ] ."],"contraindications":["4 CONTRAINDICATIONS None. None. ( 4 )"],"mechanism_of_action":["12.1 Mechanism of Action Gaucher disease is characterized by a deficiency of β-glucocerebrosidase activity, which results in accumulation of glucocerebroside in various tissues including liver, spleen, and bone marrow. The mannose sugars on imiglucerase mediate binding to and internalization by cells including macrophages. CEREZYME catalyzes the hydrolysis of glucocerebroside to glucose and ceramide."],"recent_major_changes":["Indications and Usage ( 1 ) 1/2026 Dosage and Administration ( 2.3 , 2.4 , 2.5 ) 1/2026 Warnings and Precautions ( 5.1 , 5.2 ) 1/2026"],"storage_and_handling":["Store refrigerated at 2°C to 8°C (36°F to 46°F). For storage of reconstituted and diluted solution [see Dosage and Administration (2.2) ] ."],"clinical_pharmacology":["12 CLINICAL PHARMACOLOGY 12.1 Mechanism of Action Gaucher disease is characterized by a deficiency of β-glucocerebrosidase activity, which results in accumulation of glucocerebroside in various tissues including liver, spleen, and bone marrow. The mannose sugars on imiglucerase mediate binding to and internalization by cells including macrophages. CEREZYME catalyzes the hydrolysis of glucocerebroside to glucose and ceramide. 12.2 Pharmacodynamics No formal pharmacodynamic studies have been conducted with CEREZYME. 12.3 Pharmacokinetics During one-hour intravenous infusions of four doses (7.5, 15, 30, 60 units/kg) of CEREZYME, steady-state enzymatic activity was achieved by 30 minutes. Following infusion, the half-life of plasma enzymatic activity ranged from 3.6 to 10.4 minutes. Plasma clearance ranged from 9.8 to 20.3 mL/min/kg (mean ± SD, 14.5 ± 4.0 mL/min/kg). The volume of distribution corrected for weight ranged from 0.09 to 0.15 L/kg (mean ± SD, 0.12 ± 0.02 L/kg). These variables do not appear to be influenced by dose or duration of infusion. However, only one or two patients were studied at each dose level and infusion rate. Antidrug Antibody Effects on Pharmacokinetics In patients who developed IgG antibody to CEREZYME, an apparent effect on serum enzyme levels resulted in diminished volume of distribution and clearance and increased elimination half-life compared to patients without antibody [see Adverse Reactions (6.2) ] ."],"indications_and_usage":["1 INDICATIONS AND USAGE CEREZYME is indicated for the treatment of non-central nervous system (CNS) manifestations of Type 1 or Type 3 Gaucher disease in adults and pediatric patients. CEREZYME is a hydrolytic lysosomal glucocerebrosidase-specific enzyme indicated for the treatment of non-central nervous system (CNS) manifestations of Type 1 or Type 3 Gaucher disease in adults and pediatric patients. ( 1 )"],"warnings_and_cautions":["5 WARNINGS AND PRECAUTIONS Infusion-Associated Reactions (IARs) : If a severe IAR occurs, discontinue CEREZYME immediately and initiate appropriate medical treatment. ( 5.2 ) 5.1 Hypersensitivity Reactions Including Anaphylaxis Life-threatening hypersensitivity reactions, including anaphylaxis, have been reported in patients treated with enzyme replacement therapies, including CEREZYME. In addition, other hypersensitivity reactions have included pruritus, flushing, urticaria, angioedema, chest discomfort, dyspnea, cough, cyanosis, tachycardia, and hypotension [see Adverse Reactions (6.1) ] . Patients with antibody to imiglucerase have a higher risk of hypersensitivity reactions. Conversely, not all patients with symptoms of hypersensitivity have detectable IgG antibody. Consider periodic monitoring of patients during the first year of treatment for IgG antibody formation [see Adverse Reactions (6.2) ] . Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. Administer CEREZYME under the supervision of a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis. Initiate CEREZYME in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue CEREZYME and immediately initiate appropriate medical treatment, including use of epinephrine. Consider the risks and benefits of re-administering CEREZYME following severe hypersensitivity reactions (including anaphylaxis). If the decision is made to re-administer CEREZYME, consider decreasing the infusion rate and administering antihistamines, antipyretics, and/or corticosteroids. Monitor patients for the occurrence of new signs and symptoms of a severe hypersensitivity reaction. If a mild or moderate hypersensitivity reaction occurs, consider decreasing the infusion rate, temporarily stopping the infusion, and/or administering antihistamines, antipyretics, and/or corticosteroids. Inform patients of the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis and to seek immediate medical care should symptoms occur. 5.2 Infusion-Associated Reactions Infusion-associated reactions (IARs) such as angioedema, pruritus, rash, urticaria, chest discomfort, chills, fatigue, infusion-site burning, infusion-site discomfort, infusion-site swelling, pyrexia and hypertension have been observed in patients treated with CEREZYME [see Adverse Reactions (6.1) ] . If a severe IAR occurs, discontinue CEREZYME and immediately initiate appropriate medical treatment. Consider the risks and benefits of re-administering CEREZYME following a severe IAR. If a mild or moderate IAR occurs, consider decreasing the infusion rate, temporarily stopping the infusion, and/or administering antihistamines, antipyretics, and/or corticosteroids."],"clinical_studies_table":["<table width=\"85%\"><caption>Table 3: Change from Baseline to Month 6 in Clinical Efficacy Parameters in a Randomized, Double-Blind Active-Controlled Trial of CEREZYME Compared to Alglucerase in Patients 12 Years of Age and Older with Gaucher Disease Type 1 (Study 1)</caption><col width=\"20%\" align=\"left\" valign=\"top\"/><col width=\"30%\" align=\"left\" valign=\"top\"/><col width=\"15%\" align=\"center\" valign=\"top\"/><col width=\"15%\" align=\"center\" valign=\"top\"/><col width=\"20%\" align=\"center\" valign=\"top\"/><thead><tr><th styleCode=\"Lrule Rrule\">Clinical Parameter</th><th styleCode=\"Rrule\"/><th styleCode=\"Rrule\" align=\"center\">CEREZYME   (N=15)</th><th styleCode=\"Rrule\" align=\"center\">Alglucerase   (N=15)</th><th styleCode=\"Rrule\" align=\"center\">Difference   (CEREZYME &#x2013; Alglucerase)   [95% CI]<footnote>Confidence intervals were calculated using the t distribution (appropriate for small sample sizes) and the standard error of the difference in sample means (i.e., the pooled estimate of the common standard deviation, computed as the weighted average of the standard deviations in the two treatment groups); there was no evidence that the assumption of equal variances between the groups was violated.</footnote></th></tr></thead><tbody><tr styleCode=\"Botrule\"><td styleCode=\"Lrule Rrule\" rowspan=\"2\">Hemoglobin concentration   (g/dL)</td><td styleCode=\"Rrule\">Baseline</td><td styleCode=\"Rrule\">10.7</td><td styleCode=\"Rrule\">10.9</td><td styleCode=\"Rrule\">&#x2013;</td></tr><tr styleCode=\"Botrule\"><td styleCode=\"Rrule\">Absolute Change from Baseline</td><td styleCode=\"Rrule\" align=\"center\">1.9</td><td styleCode=\"Rrule\">1.6</td><td styleCode=\"Rrule\">0.3 [-0.6, 1.3]</td></tr><tr styleCode=\"Botrule\"><td styleCode=\"Lrule Rrule\" rowspan=\"2\">Platelet count   (&#xD7; 10<sup>3</sup>/mm<sup>3</sup>)</td><td styleCode=\"Rrule\">Baseline</td><td styleCode=\"Rrule\">68.5</td><td styleCode=\"Rrule\">74.2</td><td styleCode=\"Rrule\">&#x2013;</td></tr><tr styleCode=\"Botrule\"><td styleCode=\"Rrule\">Absolute Change from Baseline</td><td styleCode=\"Rrule\" align=\"center\">22.7</td><td styleCode=\"Rrule\">15.8</td><td styleCode=\"Rrule\">6.9 [-10.4, 24.1]</td></tr><tr styleCode=\"Botrule\"><td styleCode=\"Lrule Rrule\" rowspan=\"3\">Liver volume   (mL)</td><td styleCode=\"Rrule\">Baseline</td><td styleCode=\"Rrule\">2521</td><td styleCode=\"Rrule\">2788</td><td styleCode=\"Rrule\">&#x2013;</td></tr><tr styleCode=\"Botrule\"><td styleCode=\"Rrule\">Absolute Change from Baseline</td><td styleCode=\"Rrule\" align=\"center\">-310</td><td styleCode=\"Rrule\">-307</td><td styleCode=\"Rrule\">-3 [-246, 240]</td></tr><tr styleCode=\"Botrule\"><td styleCode=\"Rrule\">Percent Change from Baseline   (%)</td><td styleCode=\"Rrule\" align=\"center\">-11</td><td styleCode=\"Rrule\">-10</td><td styleCode=\"Rrule\">-1 [-9, 7]</td></tr><tr styleCode=\"Botrule\"><td styleCode=\"Lrule Rrule\" rowspan=\"3\">Spleen volume   (mL)</td><td styleCode=\"Rrule\">Baseline</td><td styleCode=\"Rrule\">2369</td><td styleCode=\"Rrule\">2603</td><td styleCode=\"Rrule\">&#x2013;</td></tr><tr styleCode=\"Botrule\"><td styleCode=\"Rrule\">Absolute Change from Baseline</td><td styleCode=\"Rrule\" align=\"center\">-902</td><td styleCode=\"Rrule\">-874</td><td styleCode=\"Rrule\">-28 [-652, 596]</td></tr><tr><td styleCode=\"Rrule\">Percent Change from Baseline   (%)</td><td styleCode=\"Rrule\" align=\"center\">-35</td><td styleCode=\"Rrule\">-30</td><td styleCode=\"Rrule\">-5 [-14, 4]</td></tr></tbody></table>"],"nonclinical_toxicology":["13 NONCLINICAL TOXICOLOGY 13.1 Carcinogenesis, Mutagenesis, Impairment of Fertility Carcinogenesis Long-term studies in animals to evaluate carcinogenic potential have not been performed with imiglucerase. Mutagenesis Imiglucerase was negative in the Ames test. Impairment of Fertility An animal fertility study was not performed. No histopathological findings on reproductive organs were observed in 13-week toxicity studies conducted in rats and monkeys."],"adverse_reactions_table":["<table width=\"85%\"><caption>Table 2: Adverse Reactions in Adults and Pediatric Patients Treated with CEREZYME </caption><col width=\"40%\" align=\"left\" valign=\"top\"/><col width=\"60%\" align=\"left\" valign=\"top\"/><thead><tr><th styleCode=\"Lrule Rrule\"/><th styleCode=\"Rrule\">Adverse Reactions</th></tr></thead><tbody><tr styleCode=\"Botrule\"><td styleCode=\"Lrule Rrule\">Nervous system disorders</td><td styleCode=\"Rrule\">dizziness, headache</td></tr><tr styleCode=\"Botrule\"><td styleCode=\"Lrule Rrule\">Cardiac disorders</td><td styleCode=\"Rrule\">tachycardia</td></tr><tr styleCode=\"Botrule\"><td styleCode=\"Lrule Rrule\">Vascular disorders</td><td styleCode=\"Rrule\">cyanosis,<footnote ID=\"fn1\">Signs and symptoms suggestive of hypersensitivity reactions including anaphylaxis <content styleCode=\"italics\">[see <linkHtml href=\"#S5.1\">Warnings and Precautions (5.1)</linkHtml>]</content> and other infusion-associated reactions <content styleCode=\"italics\">[see <linkHtml href=\"#S5.2\">Warnings and Precautions (5.2)</linkHtml>]</content>.</footnote> flushing,<footnoteRef IDREF=\"fn1\"/> hypotension,<footnoteRef IDREF=\"fn1\"/> hypertension<footnoteRef IDREF=\"fn1\"/></td></tr><tr styleCode=\"Botrule\"><td styleCode=\"Lrule Rrule\">Respiratory, thoracic and mediastinal disorders</td><td styleCode=\"Rrule\">cough,<footnoteRef IDREF=\"fn1\"/> dyspnea,<footnoteRef IDREF=\"fn1\"/> pneumonia, pulmonary hypertension </td></tr><tr styleCode=\"Botrule\"><td styleCode=\"Lrule Rrule\">Gastrointestinal disorders</td><td styleCode=\"Rrule\">abdominal pain, diarrhea, nausea, vomiting</td></tr><tr styleCode=\"Botrule\"><td styleCode=\"Lrule Rrule\">Immune system disorders</td><td styleCode=\"Rrule\">anaphylaxis,<footnoteRef IDREF=\"fn1\"/> hypersensitivity</td></tr><tr styleCode=\"Botrule\"><td styleCode=\"Lrule Rrule\">Skin and subcutaneous tissue disorders</td><td styleCode=\"Rrule\">angioedema,<footnoteRef IDREF=\"fn1\"/> pruritus,<footnoteRef IDREF=\"fn1\"/> rash, urticaria<footnoteRef IDREF=\"fn1\"/></td></tr><tr styleCode=\"Botrule\"><td styleCode=\"Lrule Rrule\">Musculoskeletal and connective tissue disorders</td><td styleCode=\"Rrule\">back pain</td></tr><tr><td styleCode=\"Lrule Rrule\">General disorders and administration site conditions</td><td styleCode=\"Rrule\">chest discomfort,<footnoteRef IDREF=\"fn1\"/> chills, fatigue, infusion-site burning, infusion-site discomfort, infusion-site swelling, pyrexia</td></tr></tbody></table>"],"information_for_patients":["17 PATIENT COUNSELING INFORMATION Hypersensitivity Reactions Including Anaphylaxis and Infusion-Associated Reactions Advise patients and caregivers that life-threatening hypersensitivity reactions, including anaphylaxis, and IARs may occur with CEREZYME treatment. Advise patients and caregivers that anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. Inform patients and caregivers of the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis, and IARs and to seek immediate medical care should symptoms occur [see Warnings and Precautions (5.1 , 5.2) ] . Patient Registry Inform patients and caregivers that the Gaucher patient registry has been established in order to better understand the variability and progression of Gaucher disease and to continue to monitor and evaluate long-term treatment effects of CEREZYME. A pregnancy sub-registry will also monitor the effects of CEREZYME on pregnant women and their offspring [see Use in Specific Populations (8.1) ] . Encourage patients and caregivers to participate in the Gaucher patient registry. Advise patients that their participation is voluntary and may involve long-term follow-up. For information regarding the registry program, visit www.registrynxt.com or call 1-800-745-4447, extension 15500."],"spl_unclassified_section":["Manufactured by: Genzyme Corporation Cambridge, MA 02141 A SANOFI COMPANY U.S. License Number: 1596 CEREZYME is a registered trademark of Genzyme Corporation."],"dosage_and_administration":["2 DOSAGE AND ADMINISTRATION Administer CEREZYME under the supervision of a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis. ( 2.1 ) The recommended dosage is 2.5 units/kg three times a week to 60 units/kg once every two weeks administered intravenously. ( 2.2 ) Titrate the dosage based on disease severity and therapeutic goals for the patient. ( 2.2 ) See the full prescribing information for dosage modifications due to hypersensitivity reactions and/or IARs. ( 2.3 ) See the full prescribing information for preparation and administration instructions. ( 2.4 , 2.5 ) 2.1 Recommendations Prior to CEREZYME Treatment Administer CEREZYME under the supervision of a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis [see Warnings and Precautions (5.1) ] . Initiate CEREZYME in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment [see Warnings and Precautions (5.1) ] . For patients who experience hypersensitivity reactions to CEREZYME, premedicate with antihistamines and/or corticosteroids. Monitor patients for the occurrence of new hypersensitivity reactions [see Warnings and Precautions (5.1) ] . 2.2 Recommended Dosage and Administration Ensure physicians knowledgeable in the management of patients with Gaucher disease direct therapy with CEREZYME. The recommended dosage of CEREZYME is 2.5 units/kg three times a week to 60 units/kg once every two weeks administered intravenously. Titrate the dosage based on disease severity and therapeutic goals for the patient. For adults and pediatric patients weighing greater than 20 kg, infuse the diluted CEREZYME solution over 1 to 2 hours [see Dosage and Administration (2.5) ] . For pediatric patients weighing 20 kg or less, infuse the diluted CEREZYME solution over 2 hours [see Dosage and Administration (2.5) ] . 2.3 Dosage and Administration Modifications Due to Hypersensitivity Reactions and/or Infusion-Associated Reactions If a severe hypersensitivity reaction (e.g., anaphylaxis) or a severe infusion-associated reaction (IAR) occurs, discontinue CEREZYME and immediately initiate appropriate medical treatment [see Warnings and Precautions (5.1 , 5.2) ]. If a mild or moderate hypersensitivity reaction or a mild or moderate IAR occurs, consider decreasing the infusion rate, temporarily stopping the infusion, and/or administering antihistamines, antipyretics, and/or corticosteroids [ see Warnings and Precautions (5.1 , 5.2) ]. 2.4 Preparation Instructions Use aseptic technique during preparation. Reconstitution 1. Determine the number of CEREZYME vials to be reconstituted based on the individual patient's dosage regimen and remove vial(s) from the refrigerator [see Dosage and Administration (2.1) ] . 2. Reconstitute each vial of CEREZYME by slowly injecting 10.2 mL of Sterile Water for Injection, down the inside wall of each vial. 3. Roll and tilt the vial to allow the powder to dissolve completely. Visually inspect the reconstituted solution for particulate matter and discoloration. Discard if opaque particles or discoloration are observed. After reconstitution, each vial will yield CEREZYME at a concentration of 40 units/mL. 4. Withdraw the required volume of CEREZYME from the vial(s). Dilution Dilute the CEREZYME solution for infusion promptly with 0.9% Sodium Chloride Injection to a final volume that is calculated based on prescribed dose. For CEREZYME administered at a dose of 60 units/kg, use the following final volumes (see Table 1 ). For patients weighing between 1.5 kg and less than 6 kg, dilute CEREZYME to a final volume of 12 mL in a syringe for infusion. For patients weighing between 6 kg and less than 13 kg, dilute CEREZYME to a final volume of 26 mL in a syringe for infusion. For patients weighing between 13 kg and less than or equal to 20 kg, dilute CEREZYME to a final volume of 100 mL in an infusion bag. For patients weighing greater than 20 kg and less than or equal to 100 kg, dilute CEREZYME to a final volume of 200 mL in an infusion bag. For patients weighing greater than 100 kg, dilute CEREZYME to a final volume of 400 mL in an infusion bag. If CEREZYME is prescribed at a dose lower than 60 units/kg, dilute the required total units of reconstituted CEREZYME (at a concentration of 40 units/mL) with 0.9% Sodium Chloride Injection to a final concentration between 6 units/mL and 30 units/mL inclusive. If the determined dose of CEREZYME translates into a total volume of 26 mL or less, administer using a syringe for infusion via a syringe pump. For accuracy of dilution, if less than 2 mL of reconstituted CEREZYME (40 units/mL) is needed for the preparation of the determined dose, prepare a larger final volume of CEREZYME for infusion initially maintaining the final concentration of the diluted CEREZYME solution between 6 units/mL to 30 units/mL. Subsequently, discard the excess volume and administer only the volume of CEREZYME solution corresponding to the prescribed dose. Gently invert the syringe for infusion or the infusion bag to mix the solution. Throughout CEREZYME preparation, avoid vigorous shaking, agitation and foaming. Vials are for single dose only. Discard any unused solution. 2.5 Administration Instructions Visually inspect the diluted solution prior to administration of the final product for particulate matter and discoloration. Slight flocculation of protein particles (described as thin translucent fibers) may occur after dilution and does not affect the quality of the product. Administer CEREZYME as an intravenous infusion with a 0.2 micron in-line low protein-binding filter. Table 1: Total Infusion Volumes and Infusion Time for CEREZYME Patient Weight Total Infusion Volume Final concentration of diluted CEREZYME solution is between 6 units/mL and 30 units/mL. Infusion Time Maximum Infusion Rate 1.5 kg to < 6 kg 12 mL 2 hours 6 mL/hour 6 kg to < 13 kg 26 mL 2 hours 13 mL/hour 13 kg to ≤ 20 kg 100 mL 2 hours 50 mL/hour > 20 kg to ≤ 100 kg 200 mL 1 to 2 hours 200 mL/hour > 100 kg 400 mL 1 to 2 hours 400 mL/hour 2.6 Storage and Handling If the reconstituted CEREZYME vial is not used immediately: Refrigerate the reconstituted solution at 2°C to 8°C (36°F to 46°F) or store at room temperature at 20°C to 25°C (68°F to 77°F) for up to 12 hours. Refrigerate the diluted solution at 2°C to 8°C (36°F to 46°F) for up to 24 hours."],"spl_product_data_elements":["Cerezyme IMIGLUCERASE IMIGLUCERASE IMIGLUCERASE MANNITOL TRISODIUM CITRATE DIHYDRATE DISODIUM HYDROGEN CITRATE POLYSORBATE 80"],"dosage_forms_and_strengths":["3 DOSAGE FORMS AND STRENGTHS For injection: 400 units of imiglucerase as a white to off-white lyophilized powder in a single-dose vial for reconstitution. For injection: 400 units of imiglucerase as a lyophilized powder in a single-dose vial. ( 3 )"],"recent_major_changes_table":["<table width=\"100%\" styleCode=\"Noautorules\"><col width=\"70%\" align=\"left\" valign=\"middle\"/><col width=\"30%\" align=\"right\" valign=\"middle\"/><tbody><tr><td>Indications and Usage (<linkHtml href=\"#S1\">1</linkHtml>)</td><td>1/2026</td></tr><tr><td>Dosage and Administration (<linkHtml href=\"#S2.3\">2.3</linkHtml>, <linkHtml href=\"#S2.4\">2.4</linkHtml>, <linkHtml href=\"#S2.5\">2.5</linkHtml>)</td><td>1/2026</td></tr><tr><td>Warnings and Precautions (<linkHtml href=\"#S5.1\">5.1</linkHtml>, <linkHtml href=\"#S5.2\">5.2</linkHtml>)</td><td>1/2026</td></tr></tbody></table>"],"use_in_specific_populations":["8 USE IN SPECIFIC POPULATIONS 8.1 Pregnancy Pregnancy Exposure Registry There is a pregnancy exposure registry that monitors pregnancy outcomes in women exposed to CEREZYME during pregnancy. Pregnant women exposed to CEREZYME and health care providers are encouraged to contact the Gaucher patient registry at 1-800-745-4447, extension 15500 or visit www.registrynxt.com. Risk Summary Available data on more than 500 pregnancies from the international Gaucher Disease registry, postmarketing reports, published observational studies and case reports with CEREZYME or non–US-licensed imiglucerase use in pregnant women have not identified a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes. There are risks associated with symptomatic Type I Gaucher disease in pregnancy (see Clinical Considerations ) . No animal reproduction studies have been conducted with imiglucerase. The estimated background risk of major birth defects and miscarriage in the indicated population is unknown. All pregnancies have a background risk of birth defect, loss, or other adverse outcomes. In the U.S. 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. Clinical Considerations Disease-Associated Maternal and/or Embryo/Fetal Risk Pregnancy may exacerbate existing Type 1 Gaucher disease symptoms or result in new disease manifestations. Untreated symptomatic Type 1 Gaucher disease may lead to complications during pregnancy, including hepatosplenomegaly, which can interfere with the normal growth of a pregnancy and thrombocytopenia, which can lead to excessive bleeding. 8.2 Lactation Risk Summary Available published literature suggests a small amount of imiglucerase is present in breast milk immediately following an infusion of imiglucerase. Published case reports and postmarketing reports of breastfed infants have not reported adverse effects due to CEREZYME exposure. There are no data available on the effects on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for CEREZYME and any potential adverse effects on the breastfed infant from imiglucerase or from the underlying maternal condition. Lactating women with Gaucher disease treated with CEREZYME should be encouraged to enroll in the Gaucher patient registry [see Use in Specific Populations (8.1) ] . 8.4 Pediatric Use The safety and effectiveness of CEREZYME for the treatment of non-CNS manifestations of Type 1 or Type 3 Gaucher disease have been established in pediatric patients. Use of CEREZYME for the treatment of non-CNS manifestations of Type 1 Gaucher disease is supported by evidence from adequate and well-controlled trials in adults and pediatric patients 12 years of age and older and additional safety and efficacy data from an observational study of Type 1 Gaucher disease in pediatric patients [see Adverse Reactions (6.1) and Clinical Studies (14) ] . Use of CEREZYME for the treatment of non-CNS manifestations of Type 3 Gaucher disease is supported by evidence from adequate and well-controlled trials in adults and pediatric patients 12 years of age and older with Type I Gaucher disease and additional safety and efficacy data from an observational study of Type 3 Gaucher disease in adults and pediatric patients [see Adverse Reactions (6.1) and Clinical Studies (14) ] . 8.5 Geriatric Use Among the patients with Type 1 Gaucher disease in Study 3 [see Clinical Studies (14) ] who had hemoglobin or platelet count measurements 1 to 3 years after initiating CEREZYME (n = 1,053), 6% of patients were 65 and over and 1% were 75 and over. No overall differences in effectiveness of CEREZYME were observed between patients 65 years of age and older and younger adult patients with Type 1 Gaucher disease. The safety evaluation for patients 65 years of age and older with Type 1 Gaucher disease was conducted using data from a pharmacovigilance database that collected individual case safety reports and did not reveal new safety findings. Clinical studies of CEREZYME did not include sufficient numbers of patients 65 years of age and older to detect differences in safety between older and younger adult patients with Type 1 Gaucher disease. Clinical studies of CEREZYME did not include sufficient numbers of patients 65 years of age and older to detect differences in efficacy and safety between older and younger adult patients with Type 3 Gaucher disease."],"dosage_and_administration_table":["<table width=\"85%\" ID=\"table1\"><caption>Table 1: Total Infusion Volumes and Infusion Time for CEREZYME</caption><col width=\"25%\" align=\"left\" valign=\"middle\"/><col width=\"25%\" align=\"left\" valign=\"middle\"/><col width=\"25%\" align=\"left\" valign=\"middle\"/><col width=\"25%\" align=\"left\" valign=\"middle\"/><thead><tr><th styleCode=\"Lrule Rrule\" align=\"center\"><content styleCode=\"xmChange\">Patient Weight</content></th><th styleCode=\"Rrule\" align=\"center\">Total Infusion Volume<footnote>Final concentration of diluted CEREZYME solution is between 6 units/mL and 30 units/mL.</footnote></th><th styleCode=\"Rrule\" align=\"center\">Infusion Time</th><th styleCode=\"Rrule\" align=\"center\">Maximum Infusion Rate</th></tr></thead><tbody><tr styleCode=\"Botrule\"><td styleCode=\"Lrule Rrule\"><content styleCode=\"xmChange\">1.5 kg to &lt; 6 kg</content></td><td styleCode=\"Rrule\">12 mL</td><td styleCode=\"Rrule\">2 hours</td><td styleCode=\"Rrule\">6 mL/hour</td></tr><tr styleCode=\"Botrule\"><td styleCode=\"Lrule Rrule\"><content styleCode=\"xmChange\">6 kg to &lt; 13 kg</content></td><td styleCode=\"Rrule\">26 mL</td><td styleCode=\"Rrule\">2 hours</td><td styleCode=\"Rrule\">13 mL/hour</td></tr><tr styleCode=\"Botrule\"><td styleCode=\"Lrule Rrule\"><content styleCode=\"xmChange\">13 kg to &#x2264; 20 kg</content></td><td styleCode=\"Rrule\">100 mL</td><td styleCode=\"Rrule\">2 hours</td><td styleCode=\"Rrule\">50 mL/hour</td></tr><tr styleCode=\"Botrule\"><td styleCode=\"Lrule Rrule\"><content styleCode=\"xmChange\">&gt; 20 kg to &#x2264; 100 kg</content></td><td styleCode=\"Rrule\">200 mL</td><td styleCode=\"Rrule\">1 to 2 hours</td><td styleCode=\"Rrule\">200 mL/hour</td></tr><tr><td styleCode=\"Lrule Rrule\"><content styleCode=\"xmChange\">&gt; 100 kg</content></td><td styleCode=\"Rrule\">400 mL</td><td styleCode=\"Rrule\">1 to 2 hours</td><td styleCode=\"Rrule\">400 mL/hour</td></tr></tbody></table>"],"package_label_principal_display_panel":["PRINCIPAL DISPLAY PANEL - 400 Unit Vial Carton NDC 58468-4663-1 Rx only Cerezyme ® (imiglucerase) for injection 400 units per vial For intravenous infusion after reconstitution and dilution sanofi PRINCIPAL DISPLAY PANEL - 400 Unit Vial Carton"],"carcinogenesis_and_mutagenesis_and_impairment_of_fertility":["13.1 Carcinogenesis, Mutagenesis, Impairment of Fertility Carcinogenesis Long-term studies in animals to evaluate carcinogenic potential have not been performed with imiglucerase. Mutagenesis Imiglucerase was negative in the Ames test. Impairment of Fertility An animal fertility study was not performed. No histopathological findings on reproductive organs were observed in 13-week toxicity studies conducted in rats and monkeys."]},"tags":[{"label":"Hydrolytic Lysosomal Glucocerebroside-specific Enzyme","category":"class"},{"label":"Biologic","category":"modality"},{"label":"A16AB02","category":"atc"},{"label":"Intravenous","category":"route"},{"label":"Injection","category":"form"},{"label":"Active","category":"status"},{"label":"Glucosylceramide beta-glucosidase deficiency","category":"indication"},{"label":"Genzyme","category":"company"},{"label":"Approved 1990s","category":"decade"}],"phase":"marketed","safety":{"boxedWarnings":["WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. Initiate CEREZYME in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue CEREZYME and immediately initiate appropriate medical treatment, including use of epinephrine. Inform patients of the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis and to seek immediate medical care should symptoms occur [see Warnings and Precautions (5.1) ] . WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS See full prescribing information for complete boxed warning . Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. ( 5.1 ) Initiate CEREZYME in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. ( 5.1 ) If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue CEREZYME and immediately initiate appropriate medical treatment, including use of epinephrine. ( 5.1 )"],"safetySignals":[{"date":"","signal":"PYREXIA","source":"FDA FAERS","actionTaken":"209 reports"},{"date":"","signal":"FATIGUE","source":"FDA FAERS","actionTaken":"175 reports"},{"date":"","signal":"DEATH","source":"FDA FAERS","actionTaken":"173 reports"},{"date":"","signal":"PNEUMONIA","source":"FDA FAERS","actionTaken":"171 reports"},{"date":"","signal":"FALL","source":"FDA FAERS","actionTaken":"168 reports"},{"date":"","signal":"EXPOSURE DURING PREGNANCY","source":"FDA FAERS","actionTaken":"164 reports"},{"date":"","signal":"DYSPNOEA","source":"FDA FAERS","actionTaken":"148 reports"},{"date":"","signal":"BONE PAIN","source":"FDA FAERS","actionTaken":"146 reports"},{"date":"","signal":"WEIGHT INCREASED","source":"FDA FAERS","actionTaken":"146 reports"},{"date":"","signal":"MALAISE","source":"FDA FAERS","actionTaken":"135 reports"}],"commonSideEffects":[{"effect":"Hypersensitivity","drugRate":"","severity":"serious","_validated":false,"_confidence":0.3},{"effect":"Increased elimination half-life","drugRate":"reported","severity":"unknown"}],"specialPopulations":{"Lactation":"8.2 Lactation. Risk SummaryAvailable published literature suggests small amount of imiglucerase is present in breast milk immediately following an infusion of imiglucerase. Published case reports and postmarketing reports of breastfed infants have not reported adverse effects due to Cerezyme exposure. There are no data available on the effects on milk production. The developmental and health benefits of breastfeeding should be considered along with the mothers clinical need for Cerezyme and any potential adverse effects on breastfed infants or milk production.","Pregnancy":"8.1 Pregnancy. Pregnancy Exposure RegistryThere is pregnancy exposure registry that monitors pregnancy outcomes in women exposed to Cerezyme during pregnancy. Pregnant women exposed to Cerezyme and health care providers are encouraged to contact the Gaucher patient registry at 1-800-745-4447, extension 15500 or visit www.registrynxt.com.. Risk SummaryAvailable data on more than 500 pregnancies from the international Gaucher Disease registry, postmarketing reports, published observational studies and case reports with Cerezyme or non-US-licensed imiglucerase use in pregnant women have not identified drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes. There are risks associated with symptomatic Type Gaucher disease in pregnancy (see Clinical Considerations). No animal reproduction studies have been conducted with imiglucerase.The estimated background risk of major birth defects and miscarriage in the indicated population is unknown. All pregnancies have background risk of birth defect, loss, or other adverse outcomes. In the U.S. 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.. Clinical Considerations Disease-associated maternal and/or embryo/fetal riskPregnancy may exacerbate existing Type Gaucher disease symptoms or result in new disease manifestations. Untreated symptomatic Type Gaucher disease can lead to serious health consequences, including but not limited to, anemia, thrombocytopenia, bone disease, hepatomegaly or splenomegaly. The background risk of major birth defects and miscarriage in the indicated population is unknown. All pregnancies have background risk of birth defect, loss, or other adverse outcomes. In the U.S. 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.","Paediatric use":"8.4 Pediatric Use. The safety and effectiveness of Cerezyme for treatment of Type Gaucher disease that results in one or more of the following conditions: anemia, thrombocytopenia, bone disease, hepatomegaly or splenomegaly have been established in pediatric patients years of age and older. Use of Cerezyme for this indication is supported by evidence from adequate and well-controlled studies of Cerezyme and alglucerase in adults and pediatric patients 12 years of age and older, with additional data from postmarketing reports and published observational studies."}},"trials":[],"aliases":[],"company":"Sanofi","patents":[{"type":"Biologic Exclusivity","filed":"May 23, 1994","source":"FDA Purple Book","status":"Active","expires":"May 23, 2001","territory":"US","patentNumber":"BLA 020367"}],"pricing":[],"_sources":{"trials":{"url":"https://clinicaltrials.gov/search?intr=IMIGLUCERASE","method":"api_direct","source":"ClinicalTrials.gov","rawText":"","confidence":1,"sourceType":"ctgov","retrievedAt":"2026-04-20T00:32:25.999368+00:00"},"patents":{"url":"","method":"deterministic","source":"FDA Orange Book","rawText":"","confidence":1,"sourceType":"fda_orange_book","retrievedAt":"2026-04-20T00:32:25.999058+00:00"},"timeline":{"url":"https://en.wikipedia.org/wiki/Imiglucerase","method":"deterministic","source":"Wikipedia","rawText":"","confidence":0.8,"sourceType":"wikipedia","retrievedAt":"2026-04-20T00:32:33.770700+00:00"},"regulatory.ca":{"url":"","method":"api_direct","source":"Health Canada DPD","rawText":"","confidence":1,"sourceType":"health_canada_dpd","retrievedAt":"2026-04-20T00:32:32.428328+00:00"},"regulatory.eu":{"url":"","method":"api_direct","source":"European Medicines Agency","rawText":"","confidence":1,"sourceType":"ema_api","retrievedAt":"2026-04-20T00:32:26.019017+00:00"},"regulatory.us":{"url":"","method":"api_direct","source":"FDA Drugs@FDA","rawText":"","confidence":1,"sourceType":"fda_drugsfda","retrievedAt":"2026-04-20T00:32:24.602882+00:00"},"publicationCount":{"url":"https://pubmed.ncbi.nlm.nih.gov/?term=IMIGLUCERASE","method":"api_direct","source":"PubMed/NCBI","rawText":"","confidence":1,"sourceType":"pubmed","retrievedAt":"2026-04-20T00:32:33.186916+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":"2026-04-20T00:32:22.521716+00:00"},"administration.route":{"url":"","method":"deterministic","source":"FDA Label","rawText":"","confidence":1,"sourceType":"fda_label","retrievedAt":"2026-04-20T00:32:22.521760+00:00"},"safety.boxedWarnings":{"url":"","method":"deterministic","source":"FDA Label (boxed_warning)","rawText":"WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. Initiate CEREZYME in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivi","confidence":1,"sourceType":"fda_label","retrievedAt":"2026-04-20T00:32:22.521777+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":"2026-04-20T00:32:34.677320+00:00"},"mechanism.target_chembl":{"url":"","method":"api_direct","source":"ChEMBL mechanism: Glucocerebroside hydrolytic enzyme","rawText":"","confidence":1,"sourceType":"chembl","retrievedAt":"2026-04-20T00:32:33.769971+00:00"},"crossReferences.chemblId":{"url":"https://www.ebi.ac.uk/chembl/compound_report_card/CHEMBL1201632/","method":"api_direct","source":"ChEMBL (EMBL-EBI)","rawText":"","confidence":1,"sourceType":"chembl","retrievedAt":"2026-04-20T00:32:33.663204+00:00"},"regulatory.fda_application":{"url":"","method":"deterministic","source":"FDA Label","rawText":"BLA020367","confidence":1,"sourceType":"fda_label","retrievedAt":"2026-04-20T00:32:22.521786+00:00"}},"allNames":"cerezyme","offLabel":[],"synonyms":["cerezyme","imiglucerase","imiglucerase for injection","glucosylceramidase","beta-glucocerebrosidase","D-glucosyl-N-acylsphingosine glucohydrolase","acid beta-glucosidase"],"timeline":[{"date":"1994-05-23","type":"positive","source":"DrugCentral","milestone":"FDA approval (Genzyme)"},{"date":"1997-11-17","type":"positive","source":"DrugCentral","milestone":"EMA approval (Genzyme Europe B.V.)"}],"aiSummary":"Cerezyme (Imiglucerase) is a hydrolytic lysosomal glucocerebroside-specific enzyme developed by Genzyme, currently owned by the same company. It is a small molecule modality approved by the FDA in 1994 for the treatment of glucosylceramide beta-glucosidase deficiency. Cerezyme works by replacing the deficient enzyme in patients with Gaucher disease, allowing for the breakdown of glucocerebroside. The commercial status of Cerezyme is patented, and it is not yet available as a generic. Key safety considerations include monitoring for infusion reactions and hypersensitivity.","approvals":[{"date":"1994-05-23","orphan":true,"company":"GENZYME","regulator":"FDA"},{"date":"1997-11-17","orphan":false,"company":"Genzyme Europe B.V.","regulator":"EMA"}],"brandName":"Cerezyme","ecosystem":[{"indication":"Glucosylceramide beta-glucosidase deficiency","otherDrugs":[{"name":"eliglustat","slug":"eliglustat","company":"Genzyme Corp"},{"name":"miglustat","slug":"miglustat","company":"Actelion Pharms Ltd"}],"globalPrevalence":null}],"mechanism":{"novelty":"Follow-on","modality":"Biologic","drugClass":"Hydrolytic Lysosomal Glucocerebroside-specific Enzyme [EPC]","explanation":"Gaucher disease is characterized by deficiency of -glucocerebrosidase activity, which results in accumulation of glucocerebroside in various tissues including liver, spleen, and bone marrow. The mannose sugars on imiglucerase mediate binding to and internalization by cells including macrophages. Cerezyme catalyzes the hydrolysis of glucocerebroside to glucose and ceramide.","oneSentence":"Cerezyme replaces the deficient enzyme in patients with Gaucher disease, allowing for the breakdown of glucocerebroside.","technicalDetail":"Cerezyme is a recombinant form of the lysosomal enzyme glucocerebrosidase, which catalyzes the hydrolysis of glucocerebroside to glucose and ceramide, thereby facilitating the breakdown and recycling of glucocerebroside in the lysosome."},"_wikipedia":{"url":"https://en.wikipedia.org/wiki/Imiglucerase","title":"Imiglucerase","extract":"Imiglucerase is a medication used in the treatment of Gaucher's disease."},"commercial":{"launchDate":"1994","revenueYear":2024,"_launchSource":"DrugCentral (FDA 1994-05-23, GENZYME)","annualRevenue":800,"revenueSource":"Verified: Sanofi AR","revenueCurrency":"USD","revenueConfidence":"verified"},"purpleBook":{"bla":"020367","source":"FDA Purple Book","approvalDate":"May 23, 1994","licenseStatus":"Licensed","exclusivityExpiry":"May 23, 2001"},"references":[{"id":1,"url":"https://drugcentral.org/drugcard/4932","fields":["approvals","synonyms","ATC","PK","indications","contraindications","DDIs","targets","patents","FAERS"],"source":"DrugCentral"},{"id":2,"url":"https://clinicaltrials.gov/search?intr=IMIGLUCERASE","fields":["trials"],"source":"ClinicalTrials.gov"},{"id":3,"url":"https://pubmed.ncbi.nlm.nih.gov/?term=IMIGLUCERASE","fields":["publications"],"source":"PubMed/NCBI"},{"id":4,"url":"https://en.wikipedia.org/wiki/Imiglucerase","fields":["history","overview"],"source":"Wikipedia"},{"id":5,"url":"https://purplebooksearch.fda.gov/","fields":["biologicExclusivity"],"source":"FDA Purple Book"}],"_emaChecked":true,"_enrichedAt":"2026-03-30T13:55:56.772802","_validation":{"fieldsValidated":0,"lastValidatedAt":"2026-04-20T00:32:38.863334+00:00","fieldsConflicting":1,"overallConfidence":0.8},"biosimilars":[],"competitors":[{"drugName":"alglucerase","drugSlug":"alglucerase","fdaApproval":"1991-04-05","relationship":"same-class"},{"drugName":"agalsidase beta","drugSlug":"agalsidase-beta","fdaApproval":"2003-04-24","relationship":"same-class"},{"drugName":"laronidase","drugSlug":"laronidase","fdaApproval":"2003-04-30","relationship":"same-class"},{"drugName":"sacrosidase","drugSlug":"sacrosidase","fdaApproval":"1998-04-09","relationship":"same-class"},{"drugName":"alglucosidase alfa","drugSlug":"alglucosidase-alfa","fdaApproval":"2006-04-28","relationship":"same-class"},{"drugName":"galsulfase","drugSlug":"galsulfase","fdaApproval":"2005-05-31","relationship":"same-class"},{"drugName":"idursulfase","drugSlug":"idursulfase","fdaApproval":"2006-07-24","relationship":"same-class"},{"drugName":"velaglucerase alfa","drugSlug":"velaglucerase-alfa","fdaApproval":"2010-02-26","relationship":"same-class"},{"drugName":"taliglucerase alfa","drugSlug":"taliglucerase-alfa","fdaApproval":"2012-05-01","relationship":"same-class"},{"drugName":"elosulfase alfa","drugSlug":"elosulfase-alfa","fdaApproval":"2014-02-14","relationship":"same-class"},{"drugName":"asfotase alfa","drugSlug":"asfotase-alfa","fdaApproval":"2015-10-23","relationship":"same-class"},{"drugName":"sebelipase alfa","drugSlug":"sebelipase-alfa","fdaApproval":"2015-12-08","relationship":"same-class"},{"drugName":"velmanase","drugSlug":"velmanase","fdaApproval":"2023-02-16","relationship":"same-class"},{"drugName":"cerliponase alfa","drugSlug":"cerliponase-alfa","fdaApproval":"2017-04-27","relationship":"same-class"},{"drugName":"vestronidase alfa","drugSlug":"vestronidase-alfa","fdaApproval":"2017-11-15","relationship":"same-class"},{"drugName":"pegvaliase","drugSlug":"pegvaliase","fdaApproval":"2018-05-24","relationship":"same-class"},{"drugName":"pegunigalsidase alfa","drugSlug":"pegunigalsidase-alfa","fdaApproval":"2023-05-09","relationship":"same-class"},{"drugName":"avalglucosidase alfa","drugSlug":"avalglucosidase-alfa","fdaApproval":"2021-08-06","relationship":"same-class"},{"drugName":"olipudase alfa","drugSlug":"olipudase-alfa","fdaApproval":"2022-08-31","relationship":"same-class"}],"genericName":"imiglucerase","indications":{"approved":[{"name":"Glucosylceramide beta-glucosidase deficiency","source":"DrugCentral","snomedId":190794006,"regulator":"FDA","eligibility":{"Type Gaucher disease":{"age":"years of age and older","conditions":["anemia","thrombocytopenia","bone disease","hepatomegaly or splenomegaly"],"pediatric patients":"years of age and older"}}}],"offLabel":[],"pipeline":[]},"drugCategory":"active","labelChanges":[],"relatedDrugs":[{"drugId":"alglucerase","brandName":"alglucerase","genericName":"alglucerase","approvalYear":"1991","relationship":"same-class"},{"drugId":"agalsidase-beta","brandName":"agalsidase beta","genericName":"agalsidase beta","approvalYear":"2003","relationship":"same-class"},{"drugId":"laronidase","brandName":"laronidase","genericName":"laronidase","approvalYear":"2003","relationship":"same-class"},{"drugId":"sacrosidase","brandName":"sacrosidase","genericName":"sacrosidase","approvalYear":"1998","relationship":"same-class"},{"drugId":"alglucosidase-alfa","brandName":"alglucosidase alfa","genericName":"alglucosidase alfa","approvalYear":"2006","relationship":"same-class"},{"drugId":"galsulfase","brandName":"galsulfase","genericName":"galsulfase","approvalYear":"2005","relationship":"same-class"},{"drugId":"idursulfase","brandName":"idursulfase","genericName":"idursulfase","approvalYear":"2006","relationship":"same-class"},{"drugId":"velaglucerase-alfa","brandName":"velaglucerase alfa","genericName":"velaglucerase alfa","approvalYear":"2010","relationship":"same-class"},{"drugId":"taliglucerase-alfa","brandName":"taliglucerase alfa","genericName":"taliglucerase alfa","approvalYear":"2012","relationship":"same-class"},{"drugId":"elosulfase-alfa","brandName":"elosulfase alfa","genericName":"elosulfase alfa","approvalYear":"2014","relationship":"same-class"}],"trialDetails":[{"nctId":"NCT00001215","phase":"","title":"Genetic Studies of Lysosomal Storage Disorders","status":"ENROLLING_BY_INVITATION","sponsor":"National Human Genome Research Institute (NHGRI)","startDate":"1995-03-08","conditions":["Lysosomal Storage Disorders","Gaucher Disease","Parkinson Disease"],"enrollment":1000,"completionDate":""},{"nctId":"NCT04370665","phase":"NA","title":"Blood-Brain-Barrier Disruption With Cerezyme in Patient's With Parkinson's Disease","status":"COMPLETED","sponsor":"InSightec","startDate":"2020-07-16","conditions":["Parkinson Disease"],"enrollment":4,"completionDate":"2022-02-25"},{"nctId":"NCT04127578","phase":"PHASE1,PHASE2","title":"Phase 1/2a Clinical Trial of PR001 (LY3884961) in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL)","status":"ACTIVE_NOT_RECRUITING","sponsor":"Prevail Therapeutics","startDate":"2020-01-03","conditions":["Parkinson Disease"],"enrollment":32,"completionDate":"2030-12-31"},{"nctId":"NCT00302146","phase":"","title":"Positron Emission Tomography (PET) Imaging in People With Gaucher Mutations","status":"COMPLETED","sponsor":"National Human Genome Research Institute (NHGRI)","startDate":"2006-05-23","conditions":["Parkinson Disease","Gaucher Disease"],"enrollment":64,"completionDate":""},{"nctId":"NCT05565443","phase":"NA","title":"MR-guided Focused Ultrasound Plus GCase","status":"RECRUITING","sponsor":"InSightec","startDate":"2022-11-30","conditions":["Parkinsons Disease"],"enrollment":14,"completionDate":"2028-12-31"},{"nctId":"NCT03485677","phase":"PHASE3","title":"Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3","status":"COMPLETED","sponsor":"Sanofi","startDate":"2018-04-11","conditions":["Gaucher's Disease Type I","Gaucher's Disease Type III"],"enrollment":57,"completionDate":"2025-12-12"},{"nctId":"NCT00358943","phase":"","title":"International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry","status":"RECRUITING","sponsor":"Genzyme, a Sanofi Company","startDate":"1991-04-01","conditions":["Gaucher Disease","Cerebroside Lipidosis Syndrome","Glucocerebrosidase Deficiency Disease","Glucosylceramide Beta-Glucosidase Deficiency Disease"],"enrollment":12000,"completionDate":"2034-01-31"},{"nctId":"NCT04228172","phase":"","title":"Genotypic Influences on Network Progression in Parkinson's Disease","status":"ACTIVE_NOT_RECRUITING","sponsor":"Northwell Health","startDate":"2020-02-24","conditions":["Parkinson's Disease"],"enrollment":32,"completionDate":"2026-02-01"},{"nctId":"NCT04411654","phase":"PHASE1,PHASE2","title":"Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)","status":"ACTIVE_NOT_RECRUITING","sponsor":"Prevail Therapeutics","startDate":"2021-06-29","conditions":["Gaucher Disease, Type 2"],"enrollment":7,"completionDate":"2028-05"},{"nctId":"NCT05819359","phase":"PHASE2","title":"Efficacy, Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of BIA 28-6156 in GBA-PD","status":"ACTIVE_NOT_RECRUITING","sponsor":"Bial R&D Investments, S.A.","startDate":"2023-03-31","conditions":["Parkinson's Disease"],"enrollment":237,"completionDate":"2026-07-31"},{"nctId":"NCT05304195","phase":"","title":"Exploration of GCase Activity to Identify a Subpopulation Eligible for a Therapeutic Trial in Dementia With Lewy Bodies","status":"RECRUITING","sponsor":"Assistance Publique - Hôpitaux de Paris","startDate":"2023-02-17","conditions":["Dementia With Lewy Bodies"],"enrollment":236,"completionDate":"2026-06"},{"nctId":"NCT05222906","phase":"PHASE3","title":"Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3","status":"ACTIVE_NOT_RECRUITING","sponsor":"Sanofi","startDate":"2022-04-18","conditions":["Gaucher's Disease Type III"],"enrollment":43,"completionDate":"2026-10-30"},{"nctId":"NCT05518617","phase":"","title":"Molecular and Functional Imaging in Monogenic PD.","status":"RECRUITING","sponsor":"University of Exeter","startDate":"2022-07-01","conditions":["Parkinson Disease","Nervous System Disorder","Neurodegenerative Diseases","Neurodegenerative Disease, Hereditary","Parkinson's"],"enrollment":45,"completionDate":"2026-06-30"},{"nctId":"NCT04656600","phase":"PHASE4","title":"Study to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type Ⅲ","status":"COMPLETED","sponsor":"Sanofi","startDate":"2021-03-02","conditions":["Gaucher's Disease"],"enrollment":12,"completionDate":"2023-10-12"},{"nctId":"NCT05287503","phase":"PHASE2","title":"Ambroxol as a Disease-modifying Treatment in GBA-PD","status":"COMPLETED","sponsor":"Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta","startDate":"2022-03-09","conditions":["Parkinson Disease","GBA Gene Mutation"],"enrollment":65,"completionDate":"2024-12-20"},{"nctId":"NCT05529992","phase":"PHASE3","title":"A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease","status":"COMPLETED","sponsor":"Takeda","startDate":"2023-01-03","conditions":["Gaucher Disease"],"enrollment":20,"completionDate":"2024-08-05"},{"nctId":"NCT06869499","phase":"NA","title":"Study of the Prevalence of Acid Sphingomyelinase Deficiency/Niemann Pick AB and B Disease in Patients With Diffuse Interstitial Lung Disease","status":"NOT_YET_RECRUITING","sponsor":"Wladimir MAUHIN, Dr","startDate":"2025-05","conditions":["Splenomegaly","Splenectomy","Thrombopenia","Interstitial Lung Disease (ILD)","Hypocholesterolemia"],"enrollment":200,"completionDate":"2029-03-01"},{"nctId":"NCT02843035","phase":"PHASE2","title":"Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension","status":"ACTIVE_NOT_RECRUITING","sponsor":"Genzyme, a Sanofi Company","startDate":"2017-01-04","conditions":["Gaucher Disease Type 1","Gaucher Disease Type 3"],"enrollment":12,"completionDate":"2026-10-30"},{"nctId":"NCT06451419","phase":"","title":"Non Motor Symptoms in Glucocerebrosidase-related Parkinson's Disease","status":"ENROLLING_BY_INVITATION","sponsor":"Juan Pablo Romero. MD, PhD","startDate":"2024-07-01","conditions":["Parkinson Disease"],"enrollment":40,"completionDate":"2026-08-31"},{"nctId":"NCT06193421","phase":"PHASE1,PHASE2","title":"High-Dose Ambroxol in GBA1-Related Parkinson","status":"UNKNOWN","sponsor":"Agyany Pharma LTD","startDate":"2023-10-24","conditions":["Parkinson Disease","GBA Gene Mutation"],"enrollment":40,"completionDate":"2025-04-30"},{"nctId":"NCT06167603","phase":"","title":"Exploring Brain Molecular Imaging and Blood Biomarkers in Subjects With Glucocerebrosidase Mutations: Toward a Precision Medicine Approach to Characterize Parkinson's Disease Clinical Trajectories","status":"RECRUITING","sponsor":"IRCCS San Raffaele","startDate":"2023-04-30","conditions":["Parkinson Disease"],"enrollment":140,"completionDate":"2026-04-01"},{"nctId":"NCT04145037","phase":"PHASE1,PHASE2","title":"Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease","status":"TERMINATED","sponsor":"AVROBIO","startDate":"2019-05-30","conditions":["Gaucher Disease"],"enrollment":8,"completionDate":"2023-08-21"},{"nctId":"NCT05815004","phase":"PHASE2,PHASE3","title":"An Efficacy and Safety Study of AVR-RD-02 Compared to Enzyme Replacement Therapy for Treatment of Gaucher Disease Type 3","status":"WITHDRAWN","sponsor":"AVROBIO","startDate":"2023-10","conditions":["Gaucher Disease, Type 3"],"enrollment":0,"completionDate":"2027-12"},{"nctId":"NCT04120506","phase":"PHASE4","title":"Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV)","status":"COMPLETED","sponsor":"Shaare Zedek Medical Center","startDate":"2016-01-10","conditions":["Gaucher Disease, Type 1"],"enrollment":15,"completionDate":"2022-01-01"},{"nctId":"NCT03519646","phase":"NA","title":"Eliglustat on Gaucher Disease Type IIIB","status":"COMPLETED","sponsor":"National Taiwan University Hospital","startDate":"2018-04-23","conditions":["Gaucher Disease, Type III"],"enrollment":4,"completionDate":"2020-09-11"},{"nctId":"NCT02906020","phase":"PHASE2","title":"A Global Study to Assess the Drug Dynamics, Efficacy, and Safety of Venglustat (GZ/SAR402671) in Parkinson's Disease Patients Carrying a Glucocerebrosidase (GBA) Gene Mutation","status":"TERMINATED","sponsor":"Genzyme, a Sanofi Company","startDate":"2016-12-15","conditions":["Parkinson's Disease"],"enrollment":273,"completionDate":"2021-05-27"},{"nctId":"NCT02574286","phase":"PHASE4","title":"Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease","status":"COMPLETED","sponsor":"Shire","startDate":"2016-06-29","conditions":["Gaucher Disease"],"enrollment":21,"completionDate":"2020-11-30"},{"nctId":"NCT04268030","phase":"","title":"High Order Spectral Analysis of Local Field Potential Data on a Subgroup of Parkinson's Disease Patients Who Are Carriers of Mutations in the Glucocerebrosidase (GBA) Gene Undergoing DBS Electrode Placement","status":"COMPLETED","sponsor":"Rush University Medical Center","startDate":"2020-02-11","conditions":["Parkinson Disease"],"enrollment":9,"completionDate":"2020-08-30"},{"nctId":"NCT00430625","phase":"PHASE3","title":"A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease","status":"COMPLETED","sponsor":"Shire","startDate":"2007-02-15","conditions":["Gaucher Disease, Type 1"],"enrollment":25,"completionDate":"2009-04-01"},{"nctId":"NCT01614574","phase":"PHASE3","title":"Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease","status":"COMPLETED","sponsor":"Shire","startDate":"2012-03-02","conditions":["Gaucher Disease"],"enrollment":6,"completionDate":"2013-05-25"},{"nctId":"NCT00391625","phase":"PHASE1,PHASE2","title":"Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT)","status":"COMPLETED","sponsor":"Shire","startDate":"2004-09-13","conditions":["Gaucher Disease"],"enrollment":10,"completionDate":"2008-01-31"},{"nctId":"NCT01842841","phase":"PHASE3","title":"Multicenter Extension Study of Velaglucerase Alfa in Japanese Patients With Gaucher Disease","status":"COMPLETED","sponsor":"Shire","startDate":"2013-03-13","conditions":["Gaucher Disease"],"enrollment":5,"completionDate":"2014-10-08"},{"nctId":"NCT01685216","phase":"PHASE1,PHASE2","title":"Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease","status":"COMPLETED","sponsor":"Shire","startDate":"2012-09-14","conditions":["Gaucher Disease, Type 3"],"enrollment":7,"completionDate":"2015-03-15"},{"nctId":"NCT00635427","phase":"PHASE3","title":"An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease","status":"COMPLETED","sponsor":"Shire","startDate":"2008-03-13","conditions":["Gaucher Disease, Type 1"],"enrollment":95,"completionDate":"2012-12-28"},{"nctId":"NCT00478647","phase":"PHASE2,PHASE3","title":"Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase","status":"COMPLETED","sponsor":"Shire","startDate":"2007-07-25","conditions":["Gaucher Disease"],"enrollment":40,"completionDate":"2009-06-26"},{"nctId":"NCT00553631","phase":"PHASE3","title":"Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease","status":"COMPLETED","sponsor":"Shire","startDate":"2008-01-29","conditions":["Gaucher Disease, Type 1"],"enrollment":34,"completionDate":"2009-05-05"},{"nctId":"NCT00954460","phase":"","title":"Treatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher Disease","status":"APPROVED_FOR_MARKETING","sponsor":"Shire","startDate":"","conditions":["Gaucher Disease, Type 1"],"enrollment":0,"completionDate":""},{"nctId":"NCT04787887","phase":"PHASE1","title":"A Phase I Study to Compare Abcertin and EU-sourced Cerezyme® in Healthy Volunteers","status":"COMPLETED","sponsor":"ISU Abxis Co., Ltd.","startDate":"2020-01-29","conditions":["Gaucher Disease"],"enrollment":42,"completionDate":"2020-10-26"},{"nctId":"NCT03702361","phase":"PHASE4","title":"Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Treatment-naive Patients With Type 1 Gaucher Disease","status":"COMPLETED","sponsor":"Shaare Zedek Medical Center","startDate":"2018-09-04","conditions":["Primary Disease"],"enrollment":15,"completionDate":"2021-03-18"},{"nctId":"NCT03811496","phase":"","title":"Biomarker Analysis for GBA Associated Parkinson's Disease","status":"UNKNOWN","sponsor":"Lysosomal and Rare Disorders Research and Treatment Center, Inc.","startDate":"2018-02-01","conditions":["Parkinson Disease","Gaucher Disease"],"enrollment":100,"completionDate":"2020-07"},{"nctId":"NCT01132690","phase":"PHASE4","title":"A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease","status":"COMPLETED","sponsor":"Pfizer","startDate":"2010-08","conditions":["Gaucher Disease"],"enrollment":11,"completionDate":"2012-07"},{"nctId":"NCT00705939","phase":"PHASE3","title":"Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial","status":"COMPLETED","sponsor":"Pfizer","startDate":"2008-06","conditions":["Gaucher Disease"],"enrollment":45,"completionDate":"2013-08"},{"nctId":"NCT00712348","phase":"PHASE3","title":"Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase","status":"COMPLETED","sponsor":"Pfizer","startDate":"2008-12","conditions":["Gaucher Disease"],"enrollment":31,"completionDate":"2013-05"},{"nctId":"NCT00376168","phase":"PHASE3","title":"A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease","status":"COMPLETED","sponsor":"Pfizer","startDate":"2007-08","conditions":["Gaucher Disease"],"enrollment":32,"completionDate":"2009-10"},{"nctId":"NCT00962260","phase":"","title":"Expanded Access Trial of Plant Expressed Recombinant Glucocerebrosidase (prGCD) in Patients With Gaucher Disease","status":"NO_LONGER_AVAILABLE","sponsor":"Pfizer","startDate":"","conditions":["Gaucher Disease"],"enrollment":0,"completionDate":""},{"nctId":"NCT01411228","phase":"PHASE3","title":"A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease","status":"COMPLETED","sponsor":"Pfizer","startDate":"2011-09","conditions":["Gaucher Disease"],"enrollment":15,"completionDate":"2014-08"},{"nctId":"NCT02528617","phase":"PHASE4","title":"The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease","status":"WITHDRAWN","sponsor":"Baylor Research Institute","startDate":"2015-07","conditions":["Gaucher Disease Type 1","Gaucher Disease Type 3"],"enrollment":0,"completionDate":"2017-10"},{"nctId":"NCT02770625","phase":"PHASE3","title":"Phase III Study of ISU302 in Patients With Type 1 Gaucher Disease","status":"COMPLETED","sponsor":"ISU Abxis Co., Ltd.","startDate":"2011-09","conditions":["Gaucher Disease, Type 1"],"enrollment":8,"completionDate":"2014-08"},{"nctId":"NCT00244582","phase":"","title":"Convection-Enhanced Delivery of Glucocerebrosidase to Treat Type 2 Gaucher Disease","status":"COMPLETED","sponsor":"National Institute of Neurological Disorders and Stroke (NINDS)","startDate":"2005-10-22","conditions":["Type 2 Gaucher Disease"],"enrollment":1,"completionDate":"2006-11-03"},{"nctId":"NCT00001289","phase":"","title":"Effects of Enzyme Replacement in Gaucher's Disease","status":"COMPLETED","sponsor":"National Institute of Neurological Disorders and Stroke (NINDS)","startDate":"1991-09-23","conditions":["Gaucher's Disease"],"enrollment":70,"completionDate":"2008-03-03"},{"nctId":"NCT00891202","phase":"PHASE3","title":"A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)","status":"COMPLETED","sponsor":"Genzyme, a Sanofi Company","startDate":"2009-11","conditions":["Gaucher Disease, Type 1"],"enrollment":40,"completionDate":"2016-01"},{"nctId":"NCT00358150","phase":"PHASE2","title":"A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients","status":"COMPLETED","sponsor":"Genzyme, a Sanofi Company","startDate":"2006-06","conditions":["Gaucher Disease, Type 1","Cerebroside Lipidosis Syndrome","Glucocerebrosidase Deficiency Disease","Glucosylceramide Beta-Glucosidase Deficiency Disease","Gaucher Disease, Non-Neuronopathic Form"],"enrollment":26,"completionDate":"2015-12"},{"nctId":"NCT01074944","phase":"PHASE3","title":"A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE)","status":"COMPLETED","sponsor":"Genzyme, a Sanofi Company","startDate":"2010-06","conditions":["Gaucher Disease"],"enrollment":170,"completionDate":"2015-10"},{"nctId":"NCT00943111","phase":"PHASE3","title":"A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)","status":"COMPLETED","sponsor":"Genzyme, a Sanofi Company","startDate":"2009-09","conditions":["Gaucher Disease, Type 1"],"enrollment":160,"completionDate":"2015-06"},{"nctId":"NCT01344096","phase":"","title":"Thrombocytopathy in Gaucher Disease Patients","status":"UNKNOWN","sponsor":"Rabin Medical Center","startDate":"2010-10","conditions":["Gaucher Disease","Thrombocytopathy"],"enrollment":70,"completionDate":"2018-11"},{"nctId":"NCT02107846","phase":"PHASE2","title":"An Open-Label, Dose Escalation Study to Evaluate the Safety and the Pharmacokinetics of Oral PRX-112","status":"COMPLETED","sponsor":"Protalix","startDate":"2014-04","conditions":["Gaucher Disease"],"enrollment":10,"completionDate":"2015-12"},{"nctId":"NCT00001416","phase":"PHASE2","title":"Bone Response to Enzyme Replacement in Gaucher's Disease","status":"COMPLETED","sponsor":"National Institute of Neurological Disorders and Stroke (NINDS)","startDate":"1993-12","conditions":["Gaucher's Disease"],"enrollment":100,"completionDate":"2000-11"},{"nctId":"NCT01161914","phase":"PHASE3","title":"The Safety and Efficacy Study of ISU302 in Patient With Type I Gaucher Disease","status":"WITHDRAWN","sponsor":"ISU Abxis Co., Ltd.","startDate":"2011-01","conditions":["Gaucher Disease"],"enrollment":0,"completionDate":""},{"nctId":"NCT01136304","phase":"","title":"Validating a New Severity Score System for Adults With Type 1 Gaucher Disease (GD1)","status":"COMPLETED","sponsor":"University Research Foundation for Lysosomal Storage Diseases, Inc.","startDate":"2010-04","conditions":["Gaucher Disease"],"enrollment":173,"completionDate":"2013-12"},{"nctId":"NCT00364858","phase":"PHASE4","title":"Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease","status":"COMPLETED","sponsor":"Genzyme, a Sanofi Company","startDate":"2001-12","conditions":["Gaucher Disease, Type 1","Cerebroside Lipidosis Syndrome","Glucocerebrosidase Deficiency Disease","Glucosylceramide Beta-Glucosidase Deficiency Disease","Gaucher Disease, Non-Neuronopathic Form"],"enrollment":95,"completionDate":"2007-02"},{"nctId":"NCT00365131","phase":"PHASE4","title":"A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.","status":"COMPLETED","sponsor":"Genzyme, a Sanofi Company","startDate":"1997-12","conditions":["Gaucher Disease Type I","Cerebroside Lipidosis Syndrome","Clucocerebrosidase Deficiency Disease","Glucosylceramide Beta-Glucosidase Deficiency Disease","Gaucher Disease, Non-Neuronopathic Form"],"enrollment":40,"completionDate":"2004-07"},{"nctId":"NCT01747980","phase":"PHASE1","title":"Safety and Pharmacokinetics of Oral PRX-112 in Gaucher Disease Patients","status":"COMPLETED","sponsor":"Protalix","startDate":"2013-03","conditions":["Gaucher Disease"],"enrollment":16,"completionDate":"2014-03"},{"nctId":"NCT02053896","phase":"","title":"A Switch-Over Study of the Safety and Efficacy of ISU302 in Patients With Type 1 Gaucher Disease","status":"COMPLETED","sponsor":"ISU Abxis Co., Ltd.","startDate":"2011-05","conditions":["Gaucher Disease"],"enrollment":5,"completionDate":"2012-02"},{"nctId":"NCT01951989","phase":"PHASE2","title":"Intra-monocyte Imiglucerase Kinetics in Gaucher Disease","status":"UNKNOWN","sponsor":"University Hospital, Clermont-Ferrand","startDate":"2012-11","conditions":["Gaucher Disease"],"enrollment":60,"completionDate":"2016-06"},{"nctId":"NCT01881633","phase":"PHASE1","title":"A Study of the Tolerability, Safety, and Pharmacokinetics of ISU302 in Healthy Volunteers","status":"COMPLETED","sponsor":"ISU Abxis Co., Ltd.","startDate":"2010-10","conditions":["Gaucher Disease"],"enrollment":24,"completionDate":"2010-11"},{"nctId":"NCT01716741","phase":"NA","title":"Identification of Undiagnosed Gaucher Disease","status":"UNKNOWN","sponsor":"Brigham and Women's Hospital","startDate":"2012-08","conditions":["Gaucher Disease"],"enrollment":100,"completionDate":""},{"nctId":"NCT00001410","phase":"PHASE1","title":"PEG-Glucocerebrosidase for the Treatment of Gaucher Disease","status":"COMPLETED","sponsor":"National Institute of Mental Health (NIMH)","startDate":"1993-10","conditions":["Gaucher's Disease"],"enrollment":18,"completionDate":"2001-12"},{"nctId":"NCT00001234","phase":"PHASE1","title":"Gene Therapy for Gaucher's and Fabry Disease Using Viruses and Blood-Forming Cells","status":"COMPLETED","sponsor":"National Institute of Neurological Disorders and Stroke (NINDS)","startDate":"1988-01","conditions":["Gaucher's Disease"],"enrollment":120,"completionDate":"2002-04"},{"nctId":"NCT00258778","phase":"PHASE1","title":"Phase I Single Dose-Escalation Safety Study of Human Glucocerebrosidase (prGCD)","status":"COMPLETED","sponsor":"Protalix","startDate":"2005-11","conditions":["Gaucher Disease"],"enrollment":6,"completionDate":"2006-01"},{"nctId":"NCT00004293","phase":"PHASE2","title":"Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease","status":"UNKNOWN","sponsor":"National Center for Research Resources (NCRR)","startDate":"1999-11","conditions":["Gaucher's Disease"],"enrollment":24,"completionDate":""},{"nctId":"NCT00004294","phase":"PHASE1","title":"Phase I Study of Retrovirally Mediated Transfer of the Human Glucocerebrosidase Gene Into Peripheral Blood Stem Cells for Autologous Transplantation in Patients With Type I Gaucher Disease","status":"COMPLETED","sponsor":"National Center for Research Resources (NCRR)","startDate":"1999-11","conditions":["Gaucher's Disease"],"enrollment":15,"completionDate":""}],"_emaApprovals":[{"date":"1997-11-17","status":"Authorised","company":"Genzyme Europe B.V."}],"genericFilers":[],"latestUpdates":[],"manufacturing":[],"administration":{"route":"Intravenous","formulation":"Injection","formulations":[{"form":"INJECTION, POWDER, LYOPHILIZED, FOR SOLUTION","route":"INTRAVENOUS","productName":"Cerezyme"}]},"_patentsChecked":true,"crossReferences":{"MMSL":"123","NDDF":"004477","UNII":"Q6U6J48BWY","VANDF":"4020874","INN_ID":"7324","RXNORM":"84958","UMLSCUI":"C0291140","chemblId":"CHEMBL1201632","ChEMBL_ID":"CHEMBL1201632","KEGG_DRUG":"D03020","DRUGBANK_ID":"DB00053","SNOMEDCT_US":"109090004","MESH_DESCRIPTOR_UI":"D005962","MESH_SUPPLEMENTAL_RECORD_UI":"C090568"},"formularyStatus":[],"originalProduct":{"form":"INJECTION, POWDER, LYOPHILIZED, FOR SOLUTION","route":"INTRAVENOUS","company":"Genzyme Corporation","brandName":"Cerezyme","isOriginal":true,"marketingStatus":"BLA"},"_enricherVersion":"v2","developmentCodes":[],"ownershipHistory":[{"period":"1994-","companyName":"Genzyme","relationship":"Original Developer"},{"period":"1997","companyName":"Genzyme Europe B.V.","relationship":"EMA Licensee"}],"publicationCount":292,"therapeuticAreas":["Cardiovascular"],"_revenueScrapedAt":"2026-04-08 13:59:02.90133+00","atcClassification":{"source":"DrugCentral","atcCode":"A16AB02","allCodes":["A16AB02"]},"biosimilarFilings":[],"originalDeveloper":"Genzyme","recentPublications":[{"date":"2026 Mar 11","pmid":"41821052","title":"The global impact of imiglucerase therapy in children with Gaucher disease types 1 and 3: a real-world analysis from the International Collaborative Gaucher Group Gaucher Registry.","journal":"Orphanet journal of rare diseases"},{"date":"2026 Mar","pmid":"41714274","title":"Maternal and Fetal Outcomes in Imiglucerase-Treated Patients With Gaucher Disease: Real-World Evidence From the International Collaborative Gaucher Group (ICGG) Gaucher Registry Pregnancy Sub-Registry.","journal":"Journal of inherited metabolic disease"},{"date":"2006","pmid":"30000979","title":"Taliglucerase alfa.","journal":""},{"date":"2026 Feb 5","pmid":"41645321","title":"Investigating the therapeutic profile of velaglucerase alfa in paediatric patients with Gaucher disease: a systematic review across all paediatric age groups.","journal":"Orphanet journal of rare diseases"},{"date":"2026 Jan 10","pmid":"41599285","title":"Quantitative and Comparative Assessment of Recombinant Human β-Glucocerebrosidase Uptake Bioactivity Using a Stable hMMR-Expressing CHO Cell Model.","journal":"Molecules (Basel, Switzerland)"}],"companionDiagnostics":[],"genericManufacturerList":[],"status":"approved","companyName":"Sanofi","companyId":"sanofi","modality":"Enzyme","firstApprovalDate":"1994","enrichmentLevel":3,"visitCount":1,"regulatoryByCountry":[{"country_code":"US","regulator":"FDA","status":"approved","approval_date":"1994-05-23T00:00:00.000Z","mah":"GENZYME","brand_name_local":null,"application_number":""},{"country_code":"KR","regulator":"MFDS","status":"likely_approved","approval_date":"1994-05-23T00:00:00.000Z","mah":"GENZYME","brand_name_local":null,"application_number":null},{"country_code":"SA","regulator":"SFDA","status":"likely_approved","approval_date":"1994-05-23T00:00:00.000Z","mah":"GENZYME","brand_name_local":null,"application_number":null},{"country_code":"AE","regulator":"MOH","status":"likely_approved","approval_date":"1994-05-23T00:00:00.000Z","mah":"GENZYME","brand_name_local":null,"application_number":null},{"country_code":"IL","regulator":"MOH","status":"likely_approved","approval_date":"1994-05-23T00:00:00.000Z","mah":"GENZYME","brand_name_local":null,"application_number":null},{"country_code":"SG","regulator":"HSA","status":"likely_approved","approval_date":"1994-05-23T00:00:00.000Z","mah":"GENZYME","brand_name_local":null,"application_number":null},{"country_code":"US","regulator":"FDA","status":"approved","approval_date":"2014-09-05T00:00:00.000Z","mah":"GENZYME","brand_name_local":null,"application_number":"BLA020367"},{"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":"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":"EU","regulator":"EMA","status":"pending","approval_date":null,"mah":"","brand_name_local":"","application_number":""}],"trialStats":{"total":0,"withResults":0},"validation":{"fieldsValidated":0,"lastValidatedAt":"2026-04-20T00:32:38.863334+00:00","fieldsConflicting":1,"overallConfidence":0.8},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}