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BioMarin Secures Fast-Track Review For Hemophilia Gene Therapy In EU

Will It Be Second Time Lucky For Potential New Treatment?

 
• By Maureen Kenny

BioMarin plans to file for approval of its investigational hemophilia A gene therapy in the EU in June this year but its US filing is not planned until Q2 2022. It will be the company's second attempt at success in both jurisdictions.

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BioMarin is not giving up on Roctavian • Source: Alamy

BioMarin Pharmaceutical's Roctavian (valoctocogene roxaparvovec), the company's investigational gene therapy for adults with severe hemophilia A, will be fast-tracked through the EU medicines evaluation system once the company files for approval. BioMarin plans to submit a marketing authorization application to the European Medicines Agency in June.

An accelerated assessment at the EMA can shave a few months off the year or so it usually takes for an MAA to be reviewed

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