Pfizer Receives FDA Approval for Bleeding Disorder Treatment

Pfizer has received approval from the Food and Drug Administration for a rare genetic bleeding disorder treatment—the drug-maker’s first gene therapy to be approved in the U.S. The treatment, Beqvez, is for adults with moderate to severe hemophilia B—a disorder that prevents normal blood clotting—and will be available by prescription to eligible patients this quarter, according to the company. “Many people with hemophilia B struggle with the commitment and lifestyle disruption of regular FIX infusions, as well as spontaneous bleeding episodes, which can lead to painful joint damage and mobility issues,” said Adam Cuker, director of University of Pennsylvania’s Comprehensive Hemophilia and Thrombosis Program.