The Food and Drug Administration on Friday approved Pfizer 's treatment for a rare genetic bleeding disorder, making it the company's first-ever gene therapy to win clearance in the U.S. The agency greenlit the drug, which will be marketed as Beqvez, for adults with moderate to severe hemophilia B who meet certain requirements. Without that protein, called factor IX, patients with hemophilia B bruise easily and bleed more frequently and for longer periods of time. The gene therapy will compete with Australia-based CSL Behring's Hemgenix, a similar treatment that won FDA approval for hemophilia B in 2022. The company is also developing a gene therapy for Duchenne muscular dystrophy, a genetic disorder that causes muscles to weaken gradually.

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Persons: IX, hemophilia, Adam Cuker, Cuker Organizations: Drug Administration, Pfizer, U.S, Penn's, Spark Therapeutics, hemophilia Locations: U.S, Australia