Brahm Goldstein, MD, Vice President, Research and Development, Hematology at CSL Behring, discusses HEMGENIX, a pivotal gene therapy for hemophilia B.
Hemophilia B is a bleeding disorder that slows the blood clotting process, caused by genetic changes in the F9 gene. People with this disorder experience prolonged bleeding following an injury or surgery. In severe cases of hemophilia, heavy bleeding occurs after minor injury or even in the absence of injury. Serious complications can result from bleeding into the joints, muscles, brain, or other internal organs.
HEMGENIX (etranacogene dezaparvovec) is the first FDA-approved gene therapy for hemophilia B. It is an AAV5 vector administered as a one-time intravenous infusion.
The HOPE-B study was an open-label, single-dose, multi-center, multinational trial to demonstrate the efficacy and safety of etranacogene dezaparvovec. HEMGENIX met its primary endpoint of hemostatic protection over the standard of care, FIX prophylaxis. The average annualized bleeding rates were significantly reduced post-treatment by 64%. No serious adverse events were observed related to treatment.
For more information about hemophilia B and other rare hematologic disorders, visit https://checkrare.com/diseases/hematologic-disorders/