{"id":"voretigene-neparvovec-rzyl","safety":{"commonSideEffects":[{"rate":null,"effect":"Conjunctival hyperemia"},{"rate":null,"effect":"Eye pain"},{"rate":null,"effect":"Photopsia (flashing lights)"},{"rate":null,"effect":"Anterior chamber inflammation"},{"rate":null,"effect":"Cataract"}]},"_chembl":null,"_dailymed":{"setId":"08313a24-e7ce-457a-bb3f-161bc45517ee","title":"LUXTURNA (VORETIGENE NEPARVOVEC-RZYL) KIT [SPARK THERAPEUTICS, INC.]"},"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"The drug uses an adeno-associated virus (AAV) vector to introduce a working RPE65 gene into the retinal pigment epithelium. RPE65 encodes a protein essential for the visual cycle; its absence or dysfunction causes progressive vision loss. By restoring RPE65 function, the therapy enables photoreceptors to regenerate visual pigment and respond to light, thereby halting or improving vision loss.","oneSentence":"Voretigene neparvovec-rzyl is a gene therapy that delivers a functional copy of the RPE65 gene into retinal cells to restore vision in patients with inherited retinal dystrophy caused by RPE65 mutations.","_ai_confidence":"high"},"_scrapedAt":"2026-03-28T04:27:40.821Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations (Leber congenital amaurosis type 2)"}]},"trialDetails":[{"nctId":"NCT03597399","phase":"","title":"A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US","status":"COMPLETED","sponsor":"Spark Therapeutics, Inc.","startDate":"2019-01-10","conditions":"Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy","enrollment":87},{"nctId":"NCT01208389","phase":"PHASE1, PHASE2","title":"Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2","status":"ACTIVE_NOT_RECRUITING","sponsor":"Spark Therapeutics, Inc.","startDate":"2010-11","conditions":"Leber Congenital Amaurosis","enrollment":12},{"nctId":"NCT00999609","phase":"PHASE3","title":"Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis","status":"ACTIVE_NOT_RECRUITING","sponsor":"Spark Therapeutics, Inc.","startDate":"2012-10","conditions":"Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis","enrollment":31},{"nctId":"NCT03602820","phase":"","title":"Long-term Follow-up Study in Subjects Who Received Voretigene Neparvovec-rzyl (AAV2-hRPE65v2)","status":"ACTIVE_NOT_RECRUITING","sponsor":"Spark Therapeutics, Inc.","startDate":"2015-06","conditions":"Inherited Retinal Dystrophy Due to RPE65 Mutations","enrollment":41},{"nctId":"NCT00516477","phase":"PHASE1","title":"Safety Study in Subjects With Leber Congenital Amaurosis","status":"COMPLETED","sponsor":"Spark Therapeutics, Inc.","startDate":"2007-09","conditions":"Leber Congenital Amaurosis","enrollment":12}],"_emaApprovals":[],"_faersSignals":[],"_approvalHistory":[],"publicationCount":62,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":["AAV2-hRPE65v2"],"phase":"marketed","status":"active","brandName":"voretigene neparvovec-rzyl","genericName":"voretigene neparvovec-rzyl","companyName":"Spark Therapeutics, Inc.","companyId":"spark-therapeutics-inc","modality":"Biologic","firstApprovalDate":"","aiSummary":"Voretigene neparvovec-rzyl is a gene therapy that delivers a functional copy of the RPE65 gene into retinal cells to restore vision in patients with inherited retinal dystrophy caused by RPE65 mutations. Used for Inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations (Leber congenital amaurosis type 2).","enrichmentLevel":3,"visitCount":1,"trialStats":{"total":0,"withResults":0},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}