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