{"id":"alglucosidase-alfa-gz419829","safety":{"commonSideEffects":[{"rate":null,"effect":"Infusion-related reactions"},{"rate":null,"effect":"Immune response / antibody formation"},{"rate":null,"effect":"Myalgia"},{"rate":null,"effect":"Fatigue"}]},"_chembl":{"chemblId":"CHEMBL1201824","moleculeType":"Enzyme"},"_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"The drug is an enzyme replacement therapy (ERT) that provides the deficient acid alpha-glucosidase enzyme in patients with Pompe disease (glycogen storage disease type II). By replacing the missing or deficient enzyme, it enables the breakdown of lysosomal glycogen accumulation, reducing pathological glycogen storage in muscle and other tissues. This helps restore cellular function and slow disease progression.","oneSentence":"Alglucosidase alfa is a recombinant human acid alpha-glucosidase enzyme that breaks down glycogen accumulated in lysosomes in Pompe disease.","_ai_confidence":"medium"},"_scrapedAt":"2026-03-28T00:44:18.857Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Pompe disease (glycogen storage disease type II)"}]},"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":"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":"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":"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}],"_emaApprovals":[],"_faersSignals":[],"_approvalHistory":[],"publicationCount":0,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":["Myozyme","Lumizyme","Myozyme®","Lumizyme®"],"phase":"phase_3","status":"active","brandName":"Alglucosidase alfa (GZ419829)","genericName":"Alglucosidase alfa (GZ419829)","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 in Pompe disease. 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}}