Pfizer Exits Hemophilia Gene Therapy,
While CSL Behring Stands Firm with Hemgenix

In a significant shift within the hemophilia gene therapy landscape, Pfizer has announced it is discontinuing its gene therapy program for hemophilia B, pulling the plug on its FDA-approved treatment Beqvez (etranacogene dezaparvovec-drlb). The decision, announced earlier this year, stems from limited patient and physician interest, according to Pfizer’s spokesperson.

Beqvez, approved in 2024, was Pfizer’s entry into the emerging gene therapy market for hemophilia B, a rare genetic bleeding disorder affecting roughly 33,000 people in the United States. However, despite its promise, Pfizer has concluded that market demand and uptake have not met expectations, leading the company to “pause commercialization” and halt further development.

Meanwhile, CSL Behring, which markets Hemgenix (also etranacogene dezaparvovec), the first gene therapy approved by the FDA for hemophilia B in November 2022, remains committed to this innovative treatment. Though Hemgenix has treated only 12 patients in the year ending June 2024, CSL Behring’s leadership remains optimistic about the therapy’s potential.

Bob Lojewski, General Manager of CSL Behring North America, stated, “We continue to believe in the promise of Hemgenix and are focused on improving access for patients who can benefit from this transformative, one-time treatment.” He added, “We’re grateful to the hemophilia community for their ongoing support and are dedicated to advancing care options for people living with hemophilia B.”

Hemgenix offers a potentially life-changing alternative for adults with severe or moderately severe hemophilia B, providing a one-time gene therapy that can reduce or eliminate the need for frequent clotting factor infusions.

Despite the current slow uptake of gene therapies in this space, CSL Behring is actively working to raise awareness and overcome barriers to adoption. “Our goal is to ensure that patients who are eligible for Hemgenix can access it and experience the benefits of gene therapy,” said Lojewski.

With Pfizer stepping away from the hemophilia gene therapy field, CSL Behring’s Hemgenix remains a key player in this specialized market, continuing to offer hope to patients seeking new treatment options.

For more details about Hemgenix and eligibility criteria, patients and healthcare providers are encouraged to visit CSL Behring’s official website or consult with their medical specialists.

Source – Fierce