Pfizer discontinues hemophilia B treatment Beqvez
Patients, doctors show 'limited interest' in gene therapies, spokesperson says
Pfizer said it will stop development and commercialization of hemophilia B treatment Beqvez (fidanacogene elaparvovec-dzkt).
“Pfizer has made the decision to cease further development and commercialization activities with respect to Beqvez for several reasons, including the limited interest patients and their doctors have demonstrated in hemophilia gene therapies to date,” a Pfizer spokesperson said in a statement emailed to Hemophilia News Today.
Hemophilia B is caused by mutations in the gene that provides instructions to make a clotting protein called factor IX (FIX). Standard treatment involves the use of factor replacement therapies, which work to deliver a functional version of the FIX protein into the body to restore blood clotting and prevent excessive bleeding.
Beqvez is a one-time gene therapy designed to deliver a working version of the gene encoding FIX to cells in the liver, restoring the body’s ability to produce functional FIX protein. It was approved in the U.S. last year as a treatment for certain adults with moderate to severe hemophilia B. The gene therapy’s use has been authorized in Canada and in the European Union, where it has been marketed under the brand name Durveqtix.
Pfizer is communicating with hemophilia B patients who were in the process of qualifying for Beqvez treatment to discuss next steps, and the company is committed to supporting patients who have already received Beqvez as part of clinical trials, the spokesperson said. Patients looking for further information or support can contact Pfizer’s medical information line at 800-438-1985.
No plans for gene therapy
“The decisions on Pfizer’s hemophilia gene therapies do not in any way impact our commitment to people living with hemophilia,” the spokesperson said. “The Beqvez decision was not taken lightly but rather was informed by many factors specific to gene therapy.”
Pfizer has said it plans to focus more resources on Hympavzi (marstacimab-hncq), an antibody-based therapy recently approved in the U.S. and Europe to treat people with hemophilia A and hemophilia B, ages 12 and older, who don’t have inhibitors (neutralizing antibodies that can interfere with standard replacement therapies).
“People living with moderate to severe hemophilia B will continue to have multiple treatment options — including those patients who have seen successful results with Beqvez in clinical trials,” the spokesperson said. “Additionally, the recent regulatory approval and launch of Hympavzi, a first of its kind non-factor treatment option, offers an important addition to the continued availability of established … treatments that remain the cornerstone of treatments used by patients today.”
The company plans to focus on “those treatments that we believe will have the greatest impact on patients, such as Hympavzi,” the spokesperson said. The company currently has no plans to develop or market gene therapies, the person added.
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