FDA Grants Approval to Pfizer’s BEQVEZ™: A Single-Dose Gene Therapy for Hemophilia B in Adults

Today, Pfizer Inc. (NYSE: PFE) announced the approval of BEQVEZ™ (fidanacogene elaparvovec-dzkt) by the U.S. Food and Drug Administration (FDA) for the treatment of adults grappling with moderate to severe hemophilia B. This approval is particularly significant for individuals currently undergoing factor IX (FIX) prophylaxis therapy or experiencing life-threatening hemorrhages or frequent spontaneous bleeding episodes. Notably, patients must also test negative for neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid as determined by an FDA-approved test.

BEQVEZ presents a groundbreaking one-time treatment option designed to empower those with hemophilia B to generate FIX independently, thereby deviating from the current norm of regular FIX infusions, often administered multiple times a week or month. Dr. Adam Cuker, Director of the Penn Comprehensive and Hemophilia Thrombosis Program, emphasizes the potential transformative impact of BEQVEZ, alleviating both medical and treatment burdens over the long term.

Hemophilia B, characterized by a deficiency in FIX, poses challenges due to impaired blood clotting, leading to increased and prolonged bleeding. The current standard treatment involves prophylactic FIX infusions, yet many patients still face spontaneous bleeding episodes despite adherence to this regimen. The strain on healthcare systems’ resources and budgets is evident, impacting the quality of care for those with moderate to severe hemophilia B.

Acknowledging the significance of this milestone, Aamir Malik, Pfizer’s Chief U.S. Commercial Officer, highlights the company’s commitment to advancing hemophilia care through innovative solutions. Leveraging over 40 years of expertise in the field, Pfizer aims to collaborate with treatment centers, payers, and the hemophilia community to ensure BEQVEZ’s accessibility to eligible patients.

Pfizer introduces an innovative warranty program alongside BEQVEZ’s approval, offering financial protection and certainty to payers while maximizing access for eligible patients. This initiative reflects Pfizer’s dedication to enhancing patient outcomes and healthcare system sustainability.

Kim Phelan, Chief Operating Officer of The Coalition for Hemophilia B, expresses enthusiasm for BEQVEZ as a promising treatment option, offering hope for improved quality of life for eligible individuals. As BEQVEZ awaits review by the European Medicines Agency (EMA) and celebrates regulatory approval in Canada, Pfizer continues to pursue gene therapy advancements in other high-unmet-need populations, including hemophilia A and Duchenne muscular dystrophy.

BEQVEZ, an adeno-associated virus (AAV)-based gene therapy, aims to introduce a functional FIX gene variant, enabling patients to produce FIX autonomously post-treatment. Pfizer’s commitment extends beyond approval, offering personalized patient support services through the Pfizer Gene Together program, encompassing financial assistance and logistical support.

Pfizer’s licensing agreement with Spark Therapeutics underscores its dedication to spearheading pivotal studies, regulatory endeavors, and global commercialization of BEQVEZ, marking a significant stride in hemophilia B treatment innovation.

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