FDA Accepts BioMarin's BLA For Hemophilia A Gene Therapy

(RTTNews) - Shares of BioMarin Pharmaceutical Inc. (BMRN) gained nearly 5% in extended trading session Wednesday after the company announced that the U.S. Food and Drug Administration accepted its resubmission of the Biologics License Application for its investigational AAV gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A.

The Prescription Drug User Fee Act (PDUFA) target action date is March 31, 2023. If approved, valoctocogene roxaparvovec would be the first gene therapy in the U.S. for the treatment of severe hemophilia A.

"This BLA resubmission is an important step that brings us closer to delivering a gene therapy treatment choice to address the unmet needs of people with severe hemophilia A in the United States," said Hank Fuchs, President of Worldwide Research and Development at BioMarin. "In this application, we have provided a substantial body of evidence that supports the safe and effective use of valoctocogene roxaparvovec for the treatment of adults with severe hemophilia A."

BioMarin said the BLA resubmission incorporates the company's responses to all deficiencies identified in the FDA Complete Response Letter of August 18, 2020.