BioMarin Delays Resubmission, But Roctavian’s Rocky Road Winding Down

FDA Request Means Company Expects BLA By End Of September

 
• By Alaric DeArment
  
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The company had planned to refile its application at the end of June, though it is gearing up for potential approval and commercialization in Europe that could provide important experience for the US.

BioMarin said it would refile Roctavian by the end of September • Source: Shutterstock

A request for more information from the US Food and Drug Administration has once again pushed back the date when BioMarin Pharmaceutical Inc. can refile its biologics license application (BLA) for the gene therapy Roctavian (valoctocogene roxaparvovec) in hemophilia A, delaying the planned resubmission by up to three months. However, the company is better prepared to hit the market in the US as well as Europe than it was the last time around.

BioMarin released updated data on 31 May from its Phase I/II study of Roctavian showing six-year follow-up data for patients...

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