Brahm Goldstein, MD, Global Therapeutic Area Head of Hematology and Thrombosis at CSL Behring, gives an overview of hemophilia B.
As Dr. Goldstein explains, hemophilia B is a congenital bleeding disorder due to dysfunction or deficiency of coagulation Factor IX (FIX). People with this condition may bleed for longer periods of time after injury or surgery. They are also susceptible to spontaneous bleeding in muscles, joints and organs, which can be extremely painful and, in some cases, life-threatening.
Dr. Goldstein goes on to address the current standard of care for hemophilia, which includes life-long prophylactic infusions of FIX to temporarily replace or supplement low levels of the blood-clotting factor. While these infusions can significantly benefit these patients, infusions are inconvenient, painful, and can cause veins to fibrose over time, which makes the treatment more difficult. A patient’s immune system may also generate inhibitors against the replacement factor, negating the treatment benefit. Additionally, prophylactic FIX replacement therapy sometimes fails to control unobservable micro-bleeds in the joints, meaning that the degeneration can continue despite regular infusions. As such, there is a need for novel therapeutic options for hemophilia B patients.
Fortunately, positive long-term results from the phase 3 HOPE-B clinical trial evaluating etranacogene dezaparvovec (EtranaDez), an investigational adeno-associated virus five (AAV5)-based gene therapy for people living with hemophilia B were recently announced and presented at the European Association of Haemophilia and Allied Disorders (EAHAD) 2022 Annual Meeting. Overall, these results demonstrate that etranacogene dezaparvovec is statistically superior in reducing annualized bleeding rate compared to baseline FIX prophylactic therapy, even in patients with neutralizing antibodies against AAV5. Additionally, etranacogene dezaparvovec was well-tolerated and there were significant improvements in four quality-of-life domains.
To learn more about hemophilia B and other rare hematological disorders, visit checkrare.com/diseases/hematologic-disorders/