Pharmaceutical giant Pfizer has announced the discontinuation of Beqvez, its gene therapy treatment for hemophilia B, due to weak market demand. The decision comes less than a year after the therapy received FDA approval in April 2023.
Market Challenges and Strategic Shift
The company's spokesperson confirmed that limited interest from both patients and physicians in hemophilia gene therapies drove the decision to cease further development and commercialization. This move reflects broader challenges in the gene therapy market for hemophilia, where other approved treatments have also struggled to gain traction.
The gene therapy landscape for hemophilia has seen similar challenges with other products. CSL Behring's Hemgenix, priced at $3.5 million, and BioMarin's Roctavian, listed at $2.9 million, have both experienced slow adoption. BioMarin reported modest revenues of $26 million for Roctavian and has limited its sales to specific markets including the United States, Italy, and Germany.
Impact on Treatment Landscape
Despite demonstrating efficacy in providing long-term bleeding control through functional gene delivery, these advanced therapies face competition from existing effective treatments. A significant limiting factor is that patients typically have only one opportunity to receive gene therapy, which may contribute to hesitancy in adoption.
Patient Care Continuity
Pfizer has committed to maintaining support for patients who received Beqvez during clinical trials, ensuring continued monitoring through treatment sites. The company is actively communicating with patients currently in the qualification process for treatment and is in discussions with Roche, the parent company of original developer Spark Therapeutics, regarding next steps for the therapy.
Strategic Realignment
This decision represents Pfizer's complete withdrawal from viral gene replacement therapies, following earlier exits from programs in Duchenne muscular dystrophy and hemophilia A. The company plans to redirect its resources toward treatments with potentially greater patient impact, including its recently approved hemophilia A drug, Hympavzi.
The discontinuation of Beqvez follows a pattern of strategic realignment in Pfizer's rare disease portfolio, including the July 2023 abandonment of its Duchenne muscular dystrophy program and the January 2024 return of rights for a hemophilia A treatment to partner Sangamo Therapeutics.
