{"id":"alglucosidase-alfa-myozyme","safety":{"commonSideEffects":[{"rate":"10–30","effect":"Infusion-related reactions"},{"rate":"5–15","effect":"Fever"},{"rate":"5–10","effect":"Fatigue"},{"rate":"5–10","effect":"Headache"},{"rate":"5–10","effect":"Myalgia"},{"rate":null,"effect":"Immune-mediated reactions / antibody formation"}]},"_chembl":{"chemblId":"CHEMBL1201824","moleculeType":"Enzyme"},"_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. Alglucosidase alfa is a recombinant replacement enzyme that is taken up by cells via mannose-6-phosphate receptors and traffics to lysosomes, where it catalyzes glycogen degradation and reduces toxic accumulation. This enzyme replacement therapy halts or slows disease progression and improves muscle strength and function.","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":"2026-03-28T00:26:21.108Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Pompe disease (glycogen storage disease type II)"},{"name":"Infantile-onset Pompe disease"},{"name":"Late-onset Pompe disease"}]},"trialDetails":[{"nctId":"NCT04532047","phase":"PHASE1","title":"PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)","status":"RECRUITING","sponsor":"University of California, San Francisco","startDate":"2021-07-01","conditions":"MPS I, MPS II, MPS IVA","enrollment":10},{"nctId":"NCT06121011","phase":"","title":"A Global Prospective Observational Registry of Patients With Pompe Disease","status":"RECRUITING","sponsor":"Amicus Therapeutics","startDate":"2024-02-16","conditions":"Pompe Disease","enrollment":500},{"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":"NCT00567073","phase":"","title":"Pompe Pregnancy Sub-Registry","status":"RECRUITING","sponsor":"Genzyme, a Sanofi Company","startDate":"2007-06-18","conditions":"Glycogen Storage Disease Type II (GSD-II), Pompe Disease (Late-onset), Glycogenesis 2 Acid Maltase Deficiency","enrollment":20},{"nctId":"NCT03694561","phase":"","title":"Developing a Management Approach for Patients With \"Late-Onset\" Pompe Disease","status":"ACTIVE_NOT_RECRUITING","sponsor":"Duke University","startDate":"2019-03-25","conditions":"Pompe Disease, Pompe Disease (Late-onset), GAA Deficiency","enrollment":20},{"nctId":"NCT03729362","phase":"PHASE3","title":"A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease","status":"COMPLETED","sponsor":"Amicus Therapeutics","startDate":"2018-12-04","conditions":"Pompe Disease (Late-onset)","enrollment":125},{"nctId":"NCT04327973","phase":"","title":"Expanded Access for ATB200/AT2221 for the Treatment of IOPD","status":"AVAILABLE","sponsor":"Amicus Therapeutics","startDate":"","conditions":"Pompe Disease Infantile-Onset","enrollment":""},{"nctId":"NCT01380743","phase":"PHASE2","title":"Drug-drug Interaction Study","status":"COMPLETED","sponsor":"Amicus Therapeutics","startDate":"2011-10-31","conditions":"Pompe Disease","enrollment":25},{"nctId":"NCT04848779","phase":"","title":"A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)","status":"ACTIVE_NOT_RECRUITING","sponsor":"Sanofi","startDate":"2021-06-10","conditions":"Glycogen Storage Disease Type II","enrollment":16},{"nctId":"NCT01665326","phase":"","title":"Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease","status":"RECRUITING","sponsor":"Duke University","startDate":"2009-09","conditions":"Pompe Disease","enrollment":400},{"nctId":"NCT04676373","phase":"PHASE4","title":"Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen","status":"COMPLETED","sponsor":"Genzyme, a Sanofi Company","startDate":"2021-03-10","conditions":"Pompe's Disease","enrollment":41},{"nctId":"NCT06575829","phase":"PHASE4","title":"Treatment Frequency Reduction in Pompe Disease","status":"NOT_YET_RECRUITING","sponsor":"Erasmus Medical Center","startDate":"2024-10-01","conditions":"Pompe Disease (Late-onset), GAA Deficiency, Glycogen Storage Disease Type II","enrollment":10},{"nctId":"NCT02782741","phase":"PHASE3","title":"Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease","status":"COMPLETED","sponsor":"Genzyme, a Sanofi Company","startDate":"2016-11-02","conditions":"Glycogen Storage Disease Type II;Pompe's Disease","enrollment":101},{"nctId":"NCT00566878","phase":"","title":"Pompe Lactation Sub-Registry","status":"COMPLETED","sponsor":"Genzyme, a Sanofi Company","startDate":"2012-03-31","conditions":"Glycogen Storage Disease, Pompe Disease","enrollment":2},{"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":"NCT01898364","phase":"PHASE1","title":"Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.","status":"COMPLETED","sponsor":"Genzyme, a Sanofi Company","startDate":"2013-08-19","conditions":"Pompe Disease, Glycogen Storage Disease Type II (GSD II), Acid Maltase Deficiency","enrollment":24},{"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":"NCT03687333","phase":"PHASE4","title":"Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment","status":"COMPLETED","sponsor":"Genzyme, a Sanofi Company","startDate":"2018-12-04","conditions":"Glycogen Storage Disease Type II","enrollment":10},{"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":"NCT01410890","phase":"PHASE4","title":"Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease","status":"COMPLETED","sponsor":"Genzyme, a Sanofi Company","startDate":"2014-11-03","conditions":"Pompe Disease, Glycogen Storage Disease Type II (GSD II)","enrollment":21},{"nctId":"NCT05017402","phase":"","title":"Higher Dose of Alglucosidase Alpha for Pompe Disease","status":"NOT_YET_RECRUITING","sponsor":"Taipei Veterans General Hospital, Taiwan","startDate":"2021-09-01","conditions":"Glycogen Storage Disease Type II","enrollment":36},{"nctId":"NCT01710813","phase":"","title":"Alglucosidase Alfa Pompe Safety Sub-Registry","status":"COMPLETED","sponsor":"Genzyme, a Sanofi Company","startDate":"2015-03-20","conditions":"Pompe Disease","enrollment":110},{"nctId":"NCT02824068","phase":"","title":"Long-term Outcome in Late-onset Pompe Disease Treated Beyond 36 Months (STIG-Pompe-Study)","status":"COMPLETED","sponsor":"LMU Klinikum","startDate":"2016-06-01","conditions":"Pompe Disease","enrollment":112},{"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":"NCT03045042","phase":"","title":"Search for Serum/Plasma Biomarkers in Pompe's Disease","status":"UNKNOWN","sponsor":"Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau","startDate":"2017-04","conditions":"Late Onset Pompe Disease, Pompe Disease","enrollment":35},{"nctId":"NCT01597596","phase":"PHASE4","title":"A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease","status":"TERMINATED","sponsor":"Genzyme, a Sanofi Company","startDate":"2012-08","conditions":"Pompe Disease (Infantile-Onset), Glycogen Storage Disease Type II (GSD II), Glycogenosis 2","enrollment":4},{"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":"NCT00113035","phase":"","title":"Screening Protocol to Evaluate Acid Alpha-Glucosidase (GAA) Activity and GAA Gene Mutations in Patients With Late Onset Pompe Disease","status":"COMPLETED","sponsor":"Genzyme, a Sanofi Company","startDate":"2005-05","conditions":"Pompe Disease","enrollment":60},{"nctId":"NCT00158600","phase":"PHASE3","title":"A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease","status":"COMPLETED","sponsor":"Genzyme, a Sanofi Company","startDate":"2005-09","conditions":"Pompe Disease (Late-onset), Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency Disease","enrollment":90},{"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":"NCT00025896","phase":"PHASE2","title":"Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease","status":"COMPLETED","sponsor":"Genzyme, a Sanofi Company","startDate":"2001-05","conditions":"Pompe Disease, Glycogen Storage Disease Type II, Acid Maltase Deficiency Disease","enrollment":8},{"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":"NCT00455195","phase":"PHASE4","title":"Late-Onset Treatment Study Extension Protocol","status":"COMPLETED","sponsor":"Genzyme, a Sanofi Company","startDate":"2007-03","conditions":"Pompe Disease (Late-Onset), Glycogen Storage Disease Type II (GSD-II), Glycogenesis Type II","enrollment":81},{"nctId":"NCT00483379","phase":"PHASE4","title":"High Dose or High Dose Frequency Study of Alglucosidase Alfa","status":"COMPLETED","sponsor":"Genzyme, a Sanofi Company","startDate":"2007-05","conditions":"Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Glycogenesis 2 Acid Maltase Deficiency","enrollment":13},{"nctId":"NCT00520143","phase":"","title":"Alglucosidase Alfa Temporary Access Program","status":"APPROVED_FOR_MARKETING","sponsor":"Genzyme, a Sanofi Company","startDate":"","conditions":"Glycogen Storage Disease Type II (GSD-II), Pompe Disease (Late-Onset), Acid Maltase Deficiency Disease","enrollment":""},{"nctId":"NCT00125879","phase":"PHASE2, PHASE3","title":"Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602","status":"COMPLETED","sponsor":"Genzyme, a Sanofi Company","startDate":"2005-06","conditions":"Glycogen Storage Disease Type II","enrollment":16},{"nctId":"NCT00074932","phase":"NA","title":"Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-onset Pompe Disease","status":"COMPLETED","sponsor":"Genzyme, a Sanofi Company","startDate":"2004-11","conditions":"Glycogen Storage Disease Type II, Glycogenosis 2","enrollment":9},{"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},{"nctId":"NCT00250939","phase":"PHASE2","title":"A Study of rhGAA in Patients With Late-Onset Pompe Disease","status":"COMPLETED","sponsor":"Genzyme, a Sanofi Company","startDate":"2005-02","conditions":"Pompe Disease (Late-onset), Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency Disease","enrollment":5},{"nctId":"NCT00059280","phase":"PHASE2, PHASE3","title":"A Study of the Safety and Efficacy of rhGAA in Patients With Infantile-onset Pompe Disease","status":"COMPLETED","sponsor":"Genzyme, a Sanofi Company","startDate":"2003-04","conditions":"Glycogen Storage Disease Type II","enrollment":16},{"nctId":"NCT00053573","phase":"PHASE1, PHASE2","title":"rhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease)","status":"COMPLETED","sponsor":"Genzyme, a Sanofi Company","startDate":"2003-02","conditions":"Glycogen Storage Disease Type II, Pompe Disease, Acid Maltase Deficiency Disease","enrollment":20},{"nctId":"NCT00763932","phase":"PHASE2","title":"Extension Study of Long-term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) Studies","status":"COMPLETED","sponsor":"Genzyme, a Sanofi Company","startDate":"2003-04","conditions":"Pompe Disease Infantile-Onset, Glycogen Storage Disease Type II","enrollment":7},{"nctId":"NCT00051935","phase":"PHASE2","title":"A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II","status":"COMPLETED","sponsor":"Genzyme, a Sanofi Company","startDate":"2003-01","conditions":"Glycogen Storage Disease Type II, Pompe Disease, Acid Maltase Deficiency Disease","enrollment":2},{"nctId":"NCT00268944","phase":"PHASE3","title":"Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory Support","status":"COMPLETED","sponsor":"Genzyme, a Sanofi Company","startDate":"2005-12","conditions":"Pompe Disease (Late-onset), Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency Disease","enrollment":5},{"nctId":"NCT00074919","phase":"","title":"Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Infantile-onset Pompe Disease","status":"APPROVED_FOR_MARKETING","sponsor":"Genzyme, a Sanofi Company","startDate":"2003-12","conditions":"Glycogen Storage Disease Type II, Glycogenosis 2","enrollment":""},{"nctId":"NCT00765414","phase":"PHASE2","title":"Extension Study of Long-term Safety and Efficacy of Myozyme for a Single Patient With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored ERT Studies.","status":"COMPLETED","sponsor":"Genzyme, a Sanofi Company","startDate":"2003-04","conditions":"Pompe Disease Late-Onset, Glycogen Storage Disease Type II GSD II","enrollment":1}],"_emaApprovals":[],"_faersSignals":[],"_approvalHistory":[],"publicationCount":56,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":[],"phase":"marketed","status":"active","brandName":"ALGLUCOSIDASE ALFA (MYOZYME)","genericName":"ALGLUCOSIDASE ALFA (MYOZYME)","companyName":"Genzyme, a Sanofi Company","companyId":"genzyme-a-sanofi-company","modality":"Small molecule","firstApprovalDate":"","aiSummary":"Alglucosidase alfa is a recombinant human acid alpha-glucosidase enzyme that breaks down glycogen accumulated in lysosomes. Used for Pompe disease (glycogen storage disease type II).","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":0,"withResults":0},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}