FDA Approves Hemgenix, First Gene Therapy to Treat Adults With Hemophilia B
The U.S. Food and Drug Administration (FDA) approved Hemgenix (etranacogene dezaparvovec) gene therapy for the treatment of adults with hemophilia B (congenital factor 9 deficiency) who currently use factor 9 prophylaxis therapy; have current or historical life-threatening hemorrhage; or have repeated, serious spontaneous bleeding episodes.Despite advancements in the treatment of hemophilia, the prevention and treatment of bleeding episodes can adversely impact individuals’ quality of life, said Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, in a press release. “Today’s approval provides a new treatment option for patients with hemophilia B and represents important progress in the development of innovative therapies for those experiencing a high burden of disease associated with this form of hemophilia,” he said.The approval could fundamentally transform the treatment paradigm for…