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BioMarin, Spark Hemophilia Gene Therapies Progress As Accelerated Approval Favors Their Indications

 
• By Mandy Jackson

As updated Phase I/II results for BioMarin's hemophilia A and Spark's hemophilia B gene therapies were presented on May 22, it was revealed that FDA plans to make hemophilia the first gene therapy indication eligible for accelerated approval based on a surrogate endpoint.

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It remains unclear how long the effects of hemophilia gene therapies from BioMarin Pharmaceutical Inc. and Spark Therapeutics Inc. will last, but the US FDA plans to release guidance documents soon that will recommend putting gene therapies for hemophilia on course for accelerated approval.

New Phase I/II data for BioMarin's valoctocogene roxaparvovec (BMN 270) in hemophilia A and Phase I/II data for SPK-9001 in hemophilia B from Spark and partner Pfizer Inc. were presented at the World Federation of Hemophilia (WFH) World Congress in Glasgow, Scotland on May 22

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