FDA Approves Hemgenix, First Gene Therapy to Treat Adults with Hemophilia B

The US 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 advances 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 this lifelong condition, said Steven Pipe, MD, professor at the University of Michigan and a lead investigator in the HOPE-B study, in a press release from the company. “As a clinician, I look forward to being able to provide a new treatment option that may help patients treated with Hemgenix become free from the regular infusion schedule that many people living with hemophilia B rely on to protect them from the debilitating effects of the condition ,” he said.

Although there are still things we are learning about Hemgenix for hemophilia B, especially in the long term, the data that we have so far has been very encouraging, says Nigel Key, MBChB, professor at UNC School of Medicine and director of the UNC hemophilia and thrombosis center in Chapel Hill, North Carolina.