Pfizer Discontinues Hemophilia B Gene Therapy Beqvez, Signals Shift in Treatment Approach

Pfizer has announced the global discontinuation of its hemophilia B gene therapy, Beqvez, citing limited interest from patients and healthcare providers. This decision marks a significant shift in the pharmaceutical giant's approach to hemophilia treatment and raises questions about the future of gene therapies in this field.

Beqvez Withdrawal and Market Challenges

Beqvez, approved by the FDA in April 2024 with a list price of $3.5 million per dose, was positioned as a one-time treatment for hemophilia B. However, the therapy failed to gain traction in the market, leading to Pfizer's decision to halt its development and commercialization worldwide.

This move follows Pfizer's recent termination of its partnership with Sangamo Therapeutics for a hemophilia A gene therapy, giroctocogene fitelparvovec, at the end of 2024. The back-to-back withdrawals suggest a broader reassessment of Pfizer's gene therapy strategy in the hemophilia space.

Industry-Wide Challenges for Hemophilia Gene Therapies

Pfizer's retreat from hemophilia gene therapies reflects broader industry challenges. BioMarin's Roctavian, the first-ever gene therapy approved for hemophilia A in June 2023, has struggled to gain market acceptance despite its groundbreaking status. Priced at $2.9 million, Roctavian generated only $26 million in sales for 2024, prompting BioMarin to scale back its commercial efforts to focus on key markets where the therapy is approved and reimbursed.

Shifting Focus to Alternative Treatments

While moving away from gene therapies, Pfizer remains committed to hemophilia treatment through other modalities. The company will continue to invest in Hympavzi, an IgG1 monoclonal antibody designed to promote clotting. Approved by the FDA in October 2024 for both hemophilia A and B in adults and adolescents, Hympavzi offers a different treatment approach with weekly subcutaneous administration.

Similarly, Novo Nordisk is advancing its bispecific antibody, Mim8, which has shown promise in reducing bleeding events in pediatric patients. Novo Nordisk is preparing for regulatory submissions this year, supported by Phase III data demonstrating efficacy in adults and adolescents. Mim8 offers flexible dosing options, including weekly, bi-weekly, or monthly administration.

These developments suggest a potential industry pivot towards more manageable, long-acting treatments that balance efficacy with patient convenience, moving away from the high-cost, one-time treatment model of gene therapies.