BioMarin : FDA Extends Review Of BLA For Roctavian Gene Therapy For Severe Hemophilia A

(RTTNews) - BioMarin Pharmaceutical Inc. (BMRN) said Monday that it received notice from the U.S. Food and Drug Administration that the agency has extended review of the company's Biologics License Application (BLA) for Roctavian (valoctocogene roxaparvovec) gene therapy for adults with severe hemophilia A.

The FDA determined that the submission of the three-year data analysis from the ongoing Phase 3 GENEr8-1 study constituted a Major Amendment due to the substantial amount of additional data and set a new PDUFA Target Action Date of June 30, 2023.

BMRN closed Monday's regular trading at $94.30 down $6.90 or 6.82%. In the after trading, the stock further dropped $0.30 or 0.32%.

The company noted that the FDA completed a Pre-License Inspection of the manufacturing facility in early December 2022.

The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to valoctocogene roxaparvovec in March 2021. The RMAT designation is complementary to Breakthrough Therapy designation, which the company received for valoctocogene roxaparvovec in 2017.

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