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

Executive Summary

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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