- The US FDA has accepted the Biologics License Application from BioMarin Pharmaceutical ( NASDAQ: BMRN ) for its hemophilia A gene therapy candidate valoctocogene roxaparvovec.
- The FDA action date is March 31, 2023.
- The application is supported by two-year outcomes data from phase 3 GENEr8-1 study.
- The acceptance comes after BioMarin's ( BMRN ) original BLA resulted in a Complete Response letter on August 18, 2020 due to deficiencies.
- If approved, valoctocogene roxaparvovec would become the first therapy approved for severe hemophilia A.
- Earlier in October, BioMarin said it would l ay off ~4% of its workforce .
For further details see:
FDA accepts BioMarin BLA for hemophilia A gene therapy