Highlights of Valoctogene Raxaparvovec
- Valoctocogene Roxaparvovec is an investigational gene therapy treatment for Hemophilia A developed by Biomarin
- FDA issued the complete response letter requesting two years of durability data from the phase 3 study
- EMA: Biomarin withdrew MMA application marketing authorization
- Biomarin is awaiting one-year of Phase 3data for EMA resubmission and two years of Phase 3 data for FDA submission
- If approved, it will become first-in-class gene therapy for the treatment of Severe Hemophilia A
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Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A