By Stephen Nakrosis


BioMarin Pharmaceutical Inc. said Wednesday the U.S. Food and Drug Administration accepted its resubmission of a biologics license application for valoctocogene roxaparvovec, a treatment for adults with severe hemophilia A.

The company said if it is approved, valoctocogene roxaparvovec would be the first gene therapy in the U.S. for the treatment of severe hemophilia A.

Hank Fuchs, president of worldwide research and development at BioMarin, said the 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."

The company said the resubmission incorporates its responses to all deficiencies identified in the FDA complete response letter of Aug. 18, 2020.


Write to Stephen Nakrosis at stephen.nakrosis@wsj.com


(END) Dow Jones Newswires

10-12-22 1656ET