Pfizer Pulls Out of Hemophilia B Gene Therapy, Signaling a Shift in Strategy

Just weeks after ending its partnership with Sangamo Therapeutics on a separate hemophilia gene therapy, Pfizer has announced it will no longer develop or sell Beqvez, its hemophilia B gene therapy, in any global markets.

The decision, first reported by Nikkei Asia, stems from what Pfizer describes as “limited interest” from both patients and doctors in gene therapies for hemophilia. Beqvez, a one-time treatment, was approved by the FDA in April 2024 with a staggering $3.5 million price tag per dose.

This move suggests Pfizer may be backing away from hemophilia gene therapies altogether. In late 2024, the company also abandoned its partnership with Sangamo Therapeutics on giroctocogene fitelparvovec, a gene therapy for hemophilia A designed to restore the body’s ability to produce Factor VIII, a crucial clotting protein. Pfizer never provided a clear reason for dropping the project, only stating that it had decided not to move forward with regulatory or commercial plans.

A Shift Toward Alternative Hemophilia Treatments

While gene therapies may be losing favor at Pfizer, the company is still investing in hemophilia treatments. According to Reuters, the pharma giant remains committed to Hympavzi, a monoclonal antibody that helps promote blood clotting. Approved by the FDA in October 2024 for both hemophilia A and B in adults and adolescents, Hympavzi is administered weekly via subcutaneous injection.

A Larger Issue for Hemophilia Gene Therapies?

Pfizer’s exit from the gene therapy space may reflect broader challenges in the market. BioMarin’s Roctavian, the first-ever hemophilia A gene therapy, won FDA approval in June 2023 with a price tag of $2.9 million. However, the market response was underwhelming. By August 2024, BioMarin scaled back its commercial push, focusing only on the U.S., Germany, and Italy, where the treatment had both approval and reimbursement support. BioMarin’s latest earnings report revealed that Roctavian generated just $26 million in revenue in 2024—a modest return given the therapy’s high price.

Meanwhile, Novo Nordisk is taking a different approach. Earlier this month, the company reported that its bispecific antibody, Mim8, effectively reduced bleeding episodes in pediatric hemophilia patients. Novo Nordisk plans to seek regulatory approval for Mim8 this year, supported by additional Phase III data highlighting its effectiveness in adults and adolescents. Unlike one-time gene therapies, Mim8 offers flexible dosing options, with administration available once a week, every two weeks, or once a month. It seems that nothing can stop the Danish pharmaceutical giant at the moment.

Hemophilia Treatment Outlook

As Pfizer steps back from gene therapies, it’s clear the industry is still grappling with the best way to treat hemophilia. High costs, uncertain long-term efficacy, and limited patient demand seem to be hampering adoption of one-time gene therapies, while more traditional and flexible treatment options like monoclonal antibodies continue to gain traction. Whether the gene therapy model will ultimately succeed in hemophilia remains an open question—but for now, major players like Pfizer appear to be moving in a different direction.