{"id":"myozyme-alglucosidase-alfa","safety":{"commonSideEffects":[{"rate":"~50-70%","effect":"Infusion-related reactions"},{"rate":"~20-30%","effect":"Fever"},{"rate":"~10-20%","effect":"Headache"},{"rate":"~10-20%","effect":"Fatigue"},{"rate":"~10-15%","effect":"Myalgia"},{"rate":"~50-80%","effect":"Antibody formation (IgG)"}]},"_chembl":null,"_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"Pompe disease is a rare lysosomal storage disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA), leading to pathological glycogen accumulation in muscles and other tissues. Myozyme provides exogenous recombinant GAA that enters lysosomes and catalyzes glycogen breakdown, reducing toxic accumulation and improving muscle function and cardiac outcomes. This enzyme replacement therapy addresses the underlying enzymatic defect rather than just treating symptoms.","oneSentence":"Alglucosidase alfa is a recombinant human acid alpha-glucosidase enzyme that breaks down glycogen accumulated in lysosomes, restoring normal cellular function in Pompe disease.","_ai_confidence":"high"},"_scrapedAt":"2026-03-27T23:56:27.619Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Pompe disease (glycogen storage disease type II) in infants and children"},{"name":"Pompe disease in adults"}]},"trialDetails":[{"nctId":"NCT03019406","phase":"PHASE2","title":"A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa","status":"ACTIVE_NOT_RECRUITING","sponsor":"Genzyme, a Sanofi Company","startDate":"2017-10-12","conditions":"Glycogen Storage Disease Type II-Pompe's Disease","enrollment":22},{"nctId":"NCT05073783","phase":"","title":"A Study to Assess the Safety of Myozyme® and of Aldurazyme® in Male and Female Participants of Any Age Group With Pompe Disease or With Mucopolysaccharidosis Type I (MPS I) in a Home-care Setting","status":"COMPLETED","sponsor":"Sanofi","startDate":"2021-10-14","conditions":"Pompe Disease, Mucopolysaccharidosis Type I (MPS I)","enrollment":57},{"nctId":"NCT00486889","phase":"PHASE4","title":"Growth and Development Study of Alglucosidase Alfa","status":"COMPLETED","sponsor":"Genzyme, a Sanofi Company","startDate":"2008-08-26","conditions":"Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency Disease","enrollment":12},{"nctId":"NCT00701701","phase":"PHASE4","title":"Immune Tolerance Induction Study","status":"TERMINATED","sponsor":"Genzyme, a Sanofi Company","startDate":"2008-12-14","conditions":"Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Glycogenesis 2 Acid Maltase Deficiency","enrollment":4},{"nctId":"NCT02898753","phase":"PHASE1, PHASE2","title":"VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe Disease","status":"TERMINATED","sponsor":"Valerion Therapeutics, LLC","startDate":"2017-06-21","conditions":"Pompe Disease","enrollment":12},{"nctId":"NCT01526785","phase":"PHASE4","title":"A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease","status":"TERMINATED","sponsor":"Genzyme, a Sanofi Company","startDate":"2012-03","conditions":"Pompe Disease","enrollment":113},{"nctId":"NCT01288027","phase":"PHASE4","title":"Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa","status":"COMPLETED","sponsor":"Genzyme, a Sanofi Company","startDate":"2011-06","conditions":"Pompe Disease (Late-Onset), Glycogen Storage Disease Type II (GSD II), Glycogenesis 2 Acid Maltase Deficiency","enrollment":16},{"nctId":"NCT00701129","phase":"PHASE4","title":"An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease","status":"COMPLETED","sponsor":"Genzyme, a Sanofi Company","startDate":"2009-10","conditions":"Pompe Disease, Glycogen Storage Disease Type II","enrollment":4},{"nctId":"NCT00731081","phase":"","title":"Study About the Evolution of Severe Late Onset Pompe Disease Patient With Pulmonary Dysfunction and Receiving Myozyme®","status":"COMPLETED","sponsor":"Genzyme, a Sanofi Company","startDate":"2007-03","conditions":"Pompe Disease (Late-Onset), Glycogen Storage Disease Type II (GSD II), Glycogenesis 2 Acid Maltase Deficiency","enrollment":8}],"_emaApprovals":[],"_faersSignals":[],"_approvalHistory":[],"publicationCount":128,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":["Myozyme®"],"phase":"marketed","status":"active","brandName":"Myozyme® (alglucosidase alfa)","genericName":"Myozyme® (alglucosidase alfa)","companyName":"Genzyme, a Sanofi Company","companyId":"genzyme-a-sanofi-company","modality":"Biologic","firstApprovalDate":"","aiSummary":"Alglucosidase alfa is a recombinant human acid alpha-glucosidase enzyme that breaks down glycogen accumulated in lysosomes, restoring normal cellular function in Pompe disease. Used for Pompe disease (glycogen storage disease type II) in infants and children, Pompe disease in adults.","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":0,"withResults":0},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}