Pfizer Discontinues Hemophilia Gene Therapy Beqvez, Signaling Broader Industry Challenges

Pfizer's recent decision to discontinue its hemophilia B gene therapy, Beqvez, marks a significant shift in the pharmaceutical giant's portfolio and highlights ongoing challenges in the gene therapy landscape. The move comes less than a year after the FDA's approval of the one-time treatment, which carried a list price of $3.5 million per person.

Pfizer's Exit from Gene Therapy

Pfizer cited limited interest from patients and healthcare providers as a key factor in discontinuing Beqvez. The company confirmed that no patients have received the commercial treatment since its FDA approval in April 2024. This development leaves Pfizer with no active gene therapy programs, as confirmed by a company spokesperson.

The discontinuation of Beqvez is part of a larger trend for Pfizer, which has been gradually retreating from the gene therapy field. In recent months, the company has:

• Terminated a hemophilia A gene therapy partnership with Sangamo Therapeutics
• Sold preclinical gene therapy programs to AstraZeneca's Alexion in a deal worth up to $1 billion
• Abandoned a Duchenne muscular dystrophy gene therapy program following disappointing phase 3 results

Industry-Wide Struggles with Hemophilia Gene Therapies

Pfizer's experience mirrors challenges faced by other companies in the hemophilia gene therapy space:

• CSL's Hemgenix, the first-to-market hemophilia B gene therapy, has experienced a slower-than-expected launch due to complexities in the U.S. healthcare system
• BioMarin's hemophilia A gene therapy, Roctavian, has faced difficulties in sales, prompting the company to narrow its commercial focus to three markets

These setbacks highlight the broader obstacles facing gene therapies, including high costs, complex administration processes, and the need for healthcare systems to adapt to novel treatment paradigms.

Shifting Focus and Future Prospects

As Pfizer exits the gene therapy arena, the company is redirecting its hemophilia resources to Hympavzi, a once-weekly nonfactor antibody drug approved by the FDA last year for both hemophilia A and B. This pivot reflects a broader industry trend of reassessing the immediate potential of gene therapies in favor of more traditional treatment modalities.

Despite these challenges, Pfizer maintains some involvement in genetic medicine through a research collaboration with Beam Therapeutics, focusing on mRNA-delivered gene editing. However, as the four-year discovery pact enters its final year, there have been no announcements regarding potential candidates, leaving the future of this partnership uncertain.