Valoctocogene roxaparvovec

N/A

Report date: 2025/07/01 11:36:07
First In Class

Base Information

Drug NameValoctocogene roxaparvovec
Drug Alias NameAAV5-FVIII Gene Transfer-BioMarinAAV5-hFVIII-SQFactor-VIII-gene-therapyRoctavianBMN-270 +[6] 
ClassificationCell and gene therapy > Gene therapy > Viral vector-based gene therapy > Adeno-associated virus vector > AAV5 vector
Mechanisms of ActionBlood coagulation pathway activationCoagulation factor VIII modulatorsFactor VIII gene transference
TargetsCoagulation factor VIII
Therapy Area/IndicationsHemophilia ABlood Coagulation DisordersHematologic Diseases
Global Approved IndicationsHemophilia A
Approved Indications in ChinaN/A
Original Approved Country/TerritoryEU
Original Approved Date2022/08/24
Country/TerritoryBelgiumUnited StatesTaiwan +[16] 
CombinationsNo
Global Highest StatusApproved
Highest Status in ChinaN/A
Originator CompaniesBiomarin Pharmaceutical Inc
China Registration ClassN/A

Develop Information

Active CompaniesBiomarin Pharmaceutical IncBiomarin International Ltd
Inactive CompaniesN/A
Active IndicationsHemophilia A
Inactive IndicationsBlood Coagulation DisordersHematologic Diseases

Other Information

ATC CodeN/A
Related LinksN/A