HFA Responds to News of FDA Action on BioMarin Gene Therapy Application

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Today BioMarin announced that the U.S. Food and Drug Administration has issued a letter indicating that BioMarin’s investigational gene therapy for severe hemophilia A is not ready for approval in its present form. BioMarin says the FDA has asked for two years of follow-up data showing evidence of the therapy’s durability.
Many patients and their families will be disappointed to learn that gene therapy for hemophilia remains out of reach for the time being. It has been a decades-long, dearly held hope that patients could gain access to a single-dose treatment that would eliminate bleeding and improve quality of life. Always, though, HFA and the overall patient community have also insisted that health and safety remain paramount, especially in view of our community’s devastating history with tainted treatments in the 1970s and 1980s.
With health and safety as our principal touchstones, the patient community relies on the FDA to adhere to its established “gold standard” review processes and rigorous science. HFA will continue to monitor developments as BioMarin works with the FDA to provide the additional data the FDA has requested.
Read BioMarin’s full statement here.