Onasemnogene abeparvovec

N/A

Report date: 2025/11/25 14:38:19

Base Information

Drug NameOnasemnogene abeparvovec
Drug Alias NameOAV101注射液Telomeric-SMN-gene-therapy-AveXisSpinal muscular atrophy gene therapySpinal-muscular-atrophy-gene-therapy-AveXisSurvival-motor-neuron-1-gene-therapy-AveXis +[17] 
ClassificationCell and gene therapy > Gene therapy > Viral vector-based gene therapy > Adeno-associated virus vector > AAV9 vector
Mechanisms of ActionSMN gene transference
TargetsSurvival motor neuron protein
Therapy Area/IndicationsSpinal Muscular Atrophies of ChildhoodMuscular Atrophy, Spinal
Global Approved IndicationsMuscular Atrophy, Spinal
Approved Indications in ChinaN/A
Original Approved Country/TerritoryUnited States
Original Approved Date2019/05/24
Country/TerritoryBelgiumCanadaUnited States +[26] 
CombinationsNo
Global Highest StatusApproved
Highest Status in ChinaNDA/BLA
Originator CompaniesNovartis Pharma AgNovartis Gene Therapies Inc
China Registration ClassN/A

Develop Information

Active CompaniesNovartis Pharma KkNovartis Gene Therapies IncNovartis Pharma Schweiz Ag +[7] 
Inactive CompaniesN/A
Active IndicationsSpinal Muscular Atrophies of ChildhoodMuscular Atrophy, Spinal
Inactive IndicationsN/A

Other Information

ATC CodeM09AX
Related LinksWikipediaDrugBankKEGG