FDA approves gene therapy for hemophilia

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FDA approves gene therapy for hemophilia
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The FDA approved a gene therapy for hemophilia — the latest in a series of decisions to advance pricey, personalized treatments that bring new hope to patients, along with cost concerns to the health system.

The Food and Drug Administration on Tuesday approved a gene therapy for hemophilia — the latest in a series of decisions to advance pricey, personalized treatments that bring new hope to patients, along with cost concerns to the health system.With a list price of $3.5 million, Hemgenix from CSL Behring LLC will become the most expensive therapy in the world.

The treatment is given as a single dose by IV and contains a viral vector carrying a gene for the clotting factor. Its effectiveness was established based on decreases in the annualized bleeding rate of subjects in two studies. “Gene therapy for hemophilia has been on the horizon for more than two decades," said Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, adding Tuesday's decision "represents important progress in the development of innovative therapies for those experiencing a high burden of disease associated with this form of hemophilia.”

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$3.5M gene therapy for hemophilia gets FDA approval$3.5M gene therapy for hemophilia gets FDA approvalU.S. health regulators on Tuesday approved the first gene therapy for hemophilia, a $3.5 million one-time treatment for the blood-clotting disorder. The Food and Drug Administration cleared Hemgenix, an IV treatment for adults with hemophilia B, the less common form of the genetic disorder which primarily affects men. Drugmaker CSL Behring announced the $3.5 million price tag shortly after the FDA approval, saying its drug would ultimately reduce health care costs because patients would have fewer bleeding incidents and need fewer clotting treatments.
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