Pfizer Discontinues Hemophilia Gene Therapy Beqvez As Interest Wanes

According to multiple media reports on Thursday, Pfizer Inc. (NYSE:PFE) has terminated the global development and commercialization of its hemophilia B gene therapy, Beqvez.

In April 2024, the FDA approved Beqvez for moderate to severe hemophilia B in adult patients who currently use factor IX prophylaxis therapy or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes, and do not have neutralizing antibodies to adeno-associated virus serotype Rh74var capsid. 

Also Read: uniQure/ CSL Behring’s Hemophilia Gene Therapy Shows Sustained Efficacy and Safety At Four Years

In a statement to Nikkei Asia, Pfizer said the decision was due to "the limited interest patients and their doctors have demonstrated in hemophilia gene therapies.”

The U.S. drugmaker said it would focus on different treatment for the disorder, citing low interest.

In December 2024, Pfizer walked away from its hemophilia A co-development pact with Sangamo Therapeutics Inc (NASDAQ:SGMO) giroctocogene fitelparvovec, an investigational gene therapy product candidate.

According to a Reuters report, Pfizer will continue to invest resources in Hympavzi (marstacimab-hncq), which is approved for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and pediatric patients with hemophilia A or hemophilia B.

Hympavzi is the first and only anti-tissue factor pathway inhibitor (anti-TFPI) approved in the U.S. for hemophilia A or B and the first hemophilia medicine approved in the U.S. to be administered via a pre-filled, auto-injector pen.

Earlier this month, Novo Nordisk A/S (NYSE:NVO) announced interim results from the phase 3 FRONTIER3 trial of 70 children (aged 1-11 years old) with hemophilia A with and without inhibitors.

Novo Nordisk expects Mim8 regulatory submission during 2025. Data from the ongoing phase 3 FRONTIER program will be disclosed at upcoming congresses and in publications in 2025 and 2026.

In September 2024, Pfizer voluntarily withdrew all lots of Oxbryta (voxelotor) for sickle cell disease (SCD) in all markets where it is approved.

Pfizer's decision is based on the totality of clinical data indicating that the overall benefit of Oxbryta no longer outweighs the risk in the approved sickle cell patient population.

Price Action: PFE stock is up 0.87% at $26.13 at the last check on Friday.

Read Next:

• Apple Expands Apple TV+ to Android, Eyes Subscription Growth and Ecosystem Expansion, Analyst Says