Voretigene neparvovec-rzyl

N/A

Report date: 2023/10/26 16:03:26

Base Information

Drug NameVoretigene neparvovec-rzyl
Drug Alias Name奈-沃瑞替近Leber congenital amaurosis gene therapyvoretigenum neparvovecumvoretigene neparvovecLuxturna +[11] 
ClassificationCell and gene therapy > Gene therapy > Viral vector-based gene therapy > Adeno-associated virus vector > AAV2 vector
Mechanisms of ActionRPE65 gene transference
TargetsRetinoid isomerohydrolase
Therapy Area/IndicationsRetinal DystrophiesLeber Congenital AmaurosisRetinal Dysplasia +[4] 
Global Approved IndicationsRetinal DystrophiesRetinal DysplasiaVision Disorders
Approved Indications in ChinaN/A
Original Approved Country/TerritoryUnited States
Original Approved Date2017/12/19
Country/TerritoryUnited StatesEU
CombinationsNo
Global Highest StatusApproved
Highest Status in ChinaN/A
Originator CompaniesChildrens Hospital of Philadelphia
China Registration ClassN/A

Develop Information

Active CompaniesChildrens Hospital of PhiladelphiaSpark Therapeutics IncNovartis Europharm Ltd
Inactive CompaniesN/A
Active IndicationsRetinal DystrophiesLeber Congenital AmaurosisRetinal Dysplasia +[4] 
Inactive IndicationsN/A

Other Information

ATC CodeS01
Related LinksN/A