- BioMarin Pharmaceutical ( NASDAQ: BMRN ) on Thursday said it had resubmitted its biologics license application (BLA) to the U.S. FDA for its gene therapy valoctocogene roxaparvovec for the treatment of severe hemophilia A.
- The FDA had previously rejected the BLA in Aug. 2020 , citing the need for additional two-year data from an ongoing phase 3 study.
- In Thursday's statement , BMRN said the resubmitted BLA incorporated two-year outcomes from the phase 3 study and supporting data from five years of follow-up from an ongoing phase 1/2 dose escalation study.
- The company expects a response from the U.S. drug regulator by the end of Oct.
- If approved, valoctocogene roxaparvovec would be the first commercially-available gene therapy in the U.S. for the treatment of severe hemophilia A, BMRN said.
- BioMarin ( BMRN ) stock earlier closed -0.9% at $85.06.
For further details see:
BioMarin resubmits biologics license application to FDA for hemophilia A gene therapy