{"id":"onasemnogene-abeparvovec-xioi","safety":{"commonSideEffects":[{"rate":null,"effect":"Elevated transaminases (ALT/AST)"},{"rate":null,"effect":"Thrombocytopenia"},{"rate":null,"effect":"Hepatotoxicity"},{"rate":null,"effect":"Fever"}]},"_chembl":{"chemblId":"CHEMBL4297240","moleculeType":"Gene"},"_dailymed":{"setId":"68cd4f06-70e1-40d8-bedb-609ec0afa471","title":"ZOLGENSMA (ONASEMNOGENE ABEPARVOVEC-XIOI) KIT [NOVARTIS GENE THERAPIES, INC.]"},"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"The drug uses an AAV9 vector to cross the blood-brain barrier and deliver the SMN1 gene directly to motor neurons and other tissues. Once delivered, the functional SMN1 gene is expressed, producing survival motor neuron (SMN) protein, which is deficient or absent in spinal muscular atrophy patients due to mutations in the endogenous SMN1 gene. This protein restoration halts or reverses the progressive motor neuron degeneration characteristic of the disease.","oneSentence":"Onasemnogene abeparvovec-xioi is a gene therapy that delivers a functional copy of the SMN1 gene via an adeno-associated virus vector to restore survival motor neuron protein production in patients with spinal muscular atrophy.","_ai_confidence":"high"},"_scrapedAt":"2026-03-28T01:24:15.577Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Spinal muscular atrophy (SMA) in pediatric patients"}]},"trialDetails":[{"nctId":"NCT07047144","phase":"PHASE2","title":"A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy","status":"RECRUITING","sponsor":"Scholar Rock, Inc.","startDate":"2025-09-15","conditions":"Spinal Muscular Atrophy, SMA, Spinal Muscular Atrophy Type 2","enrollment":52},{"nctId":"NCT03837184","phase":"PHASE3","title":"Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies","status":"COMPLETED","sponsor":"Novartis Gene Therapies","startDate":"2019-05-31","conditions":"Spinal Muscular Atrophy Type I","enrollment":2},{"nctId":"NCT03306277","phase":"PHASE3","title":"Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1","status":"COMPLETED","sponsor":"Novartis Gene Therapies","startDate":"2017-10-24","conditions":"SMA - Spinal Muscular Atrophy, Gene Therapy","enrollment":22},{"nctId":"NCT03505099","phase":"PHASE3","title":"Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2","status":"COMPLETED","sponsor":"Novartis Gene Therapies","startDate":"2018-04-02","conditions":"Spinal Muscular Atrophy","enrollment":30},{"nctId":"NCT03461289","phase":"PHASE3","title":"Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1","status":"COMPLETED","sponsor":"Novartis Gene Therapies","startDate":"2018-08-16","conditions":"SMA","enrollment":33},{"nctId":"NCT03381729","phase":"PHASE1","title":"Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy","status":"TERMINATED","sponsor":"Novartis Gene Therapies","startDate":"2017-12-14","conditions":"Spinal Muscular Atrophy","enrollment":32},{"nctId":"NCT03421977","phase":"","title":"Long-Term Follow-up Study for Patients From AVXS-101-CL-101","status":"ACTIVE_NOT_RECRUITING","sponsor":"Novartis Gene Therapies","startDate":"2017-09-21","conditions":"Spinal Muscular Atrophy 1","enrollment":13},{"nctId":"NCT04042025","phase":"PHASE3","title":"Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi","status":"ACTIVE_NOT_RECRUITING","sponsor":"Novartis Gene Therapies","startDate":"2020-02-10","conditions":"Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III","enrollment":85}],"_emaApprovals":[],"_faersSignals":[{"count":3,"reaction":"DRUG INEFFECTIVE"},{"count":2,"reaction":"FATIGUE"},{"count":1,"reaction":"ASPIRATION"},{"count":1,"reaction":"ASTHENIA"},{"count":1,"reaction":"DECREASED APPETITE"},{"count":1,"reaction":"DYSPHAGIA"},{"count":1,"reaction":"INFLAMMATORY MARKER INCREASED"},{"count":1,"reaction":"LIVER INJURY"},{"count":1,"reaction":"MUSCULAR WEAKNESS"},{"count":1,"reaction":"NECROTISING ENTEROCOLITIS NEONATAL"}],"_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":["Zolgensma","AVXS-101","OAV101"],"phase":"marketed","status":"active","brandName":"Onasemnogene Abeparvovec-xioi","genericName":"Onasemnogene Abeparvovec-xioi","companyName":"Novartis Gene Therapies","companyId":"novartis-gene-therapies","modality":"Biologic","firstApprovalDate":"","aiSummary":"Onasemnogene abeparvovec-xioi is a gene therapy that delivers a functional copy of the SMN1 gene via an adeno-associated virus vector to restore survival motor neuron protein production in patients with spinal muscular atrophy. Used for Spinal muscular atrophy (SMA) in pediatric patients.","enrichmentLevel":3,"visitCount":1,"trialStats":{"total":6,"withResults":4},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}