FDA Action Date on BioMarin’s Roctavian Delayed Due to ‘Substantial’ Data

The FDA has extended its decision date from March 31 to June 30 on BioMarin Pharmaceutical’s biologics license application (BLA) for Roctavian (valoctocogene roxaparvovec), an investigational gene therapy for patients with severe hemophilia A.

The agency’s decision was based on the company’s submission of three-year analysis from an ongoing phase 3 study, which the FDA considers a “substantial amount of additional data,” and a major amendment.

BioMarin has several clinical studies underway to evaluate the gene therapy for treatment of hemophilia A, the company said.

The gene therapy previously received the FDA’s Regenerative Medicine Advanced Therapy designation in 2021 and Breakthrough Therapy status in 2017. Roctavian has also received conditional marketing authorization in the EU.