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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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  • I don’t understand this decision at all. Since when does the durability of a medication impact whether it is approved? This is a vast improvement over what it currently available, and can be fine-tuned as time goes on. I am appalled at what is going on these days at the FDA – their decisions are perplexing and disturbing. I hope they will reconsider.

  • As a retired physician I am very surprised and disappointed by the FDA decision. It would not take much factor V111 improvement to let hemophiliacs have a much better quality of life. I hope this heartless decision can be overturned!!

  • It doesn’t take much factor VIII to prevent bleeding. Those hemophiliacs who bleed enough to require the 300,000 dollars a year infusions of recombinant factor VIII tend to natively have less than 1 percent (of average normal) factor VIII. If they have as much as 5 percent, they tend not to bleed. Keeping the level above 10 percent would almost certainly prevent them from bleeding at all. Good luck to BioMarin. If this gene therapy works it would be a God Send to a lot of people. These bad hemophiliacs require iv infusion three times a week, 52 weeks a year. The cost may be as high as 500,000 dollars a year and they still get joint bleeds from time to time. Think of the difference the gene therapy could make in their quality of life.

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