Pfizer’s BEQVEZ, a one-time gene therapy, has received FDA approval for treating adults with moderate to severe hemophilia B. Patients who have life-threatening hemorrhages, repeated spontaneous bleeding episodes, or current use of factor IX (FIX) prophylaxis therapy are eligible for this treatment.
Hemophilia B is a rare disorder that affects blood clotting due to a deficiency in FIX. The standard treatment involves frequent intravenous FIX infusions. However, BEQVEZ aims to eliminate this by enabling patients to produce FIX on their own, reducing the need for regular injections.
Dr. Adam Cuker, Director of the Penn Comprehensive and Hemophilia Thrombosis Program, highlights the potential of BEQVEZ to alleviate the burden and disruption associated with traditional FIX infusions.
BEQVEZ’s unique mechanism may also benefit healthcare systems by reducing strain on resources and budgets. Despite prophylactic infusions, many hemophilia B patients experience spontaneous bleeding episodes.
Pfizer’s Chief U.S. Commercial Officer, Aamir Malik, emphasizes the company’s commitment to advancing hemophilia care. Pfizer plans to collaborate with healthcare providers and the hemophilia community to ensure accessibility to BEQVEZ for eligible patients.
For patients, BEQVEZ offers not only long-term protection against bleeding but also potential time savings from eliminated treatments. Kim Phelan, Chief Operating Officer of The Coalition for Hemophilia B, highlights the positive impact BEQVEZ could have on the lives of hemophilia B patients.
Pfizer has implemented a warranty program to assure durability of treatment response. This measure provides certainty for payers, maximizes access for eligible patients, and offers financial protection against inefficacy risks.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
