The Food and Drug Administration on Wednesday rejected a one-time gene therapy for hemophilia A, delaying what would have been a major medical milestone in a decision that upended the expectations of doctors, patients, and Wall Street.
The FDA’s move means the gene therapy, which promised to revolutionize the treatment of the bleeding disorder, will be delayed until at least 2022, according to BioMarin, the treatment’s manufacturer.
In clinical trials, BioMarin’s gene therapy demonstrated a 95% reduction in the bleeding episodes that characterize hemophilia A. Over the course of four years, patients needed 96% fewer doses of costly medicines that improve blood clotting.
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