Approved: First Gene Therapy for Hemophilia B

(RxWiki News) There’s a new treatment for a serious bleeding disorder.

The US Food and Drug Administration (FDA) recently approved Hemgenix (etranacogene dezaparvovec) to treat hemophilia B.

This treatment is a gene therapy meant for “adults with Hemophilia B (congenital Factor IX deficiency) who currently use Factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes,” according to the FDA in a press release.

Health officials said this new approval was an innovative step forward in treatments for health conditions like hemophilia.

“Gene therapy for hemophilia has been on the horizon for more than two decades,” said Dr. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, in a press release. “Despite advancements in the treatment of hemophilia, the prevention and treatment of bleeding episodes can adversely impact individuals’ quality of life.”

Hemophilia B is a genetic disorder that causes patients to bleed. And bleeding that occurs over long periods of time can be dangerous and lead to serious complications such bleeding into the organs.

Patients’ blood does not clot properly because it is missing a certain protein.

Typically, those with severe Hemophilia B are treated with ongoing intravenous infusions of the missing protein to encourage blood clotting. 

Hemgenix, on the other hand, is a one-time therapy that, according to the FDA, encourages the body to produce the missing protein necessary for proper blood clotting. This limits episodes of bleeding. This new therapy is administered into the vein.

While being studied, those patients who received Hemgenix, saw reductions in bleeding based on annualized bleeding rate (ABR).

Side effects of Hemgenix included flu-like symptoms, elevated liver enzymes, mild infusion-related reactions and headaches.

The FDA approved Hemgenix for CSL Behring LLC.