Pfizer Halts Commercialization of FDA-Approved Hemophilia B Gene Therapy Beqvez Amid Limited Market Interest

Recently, Pfizer announced that less than a year after the launch of its hemophilia B gene therapy drug fidanacogene elaparvovec (marketed as Beqvez in the U.S. and Durveqtix in Europe), it has decided to halt the development and commercialization of the product.

Pfizer said in a statement released on Friday that "the current interest in hemophilia gene therapy among patients and doctors is limited."

Beqvez is a one-time treatment drug, originally developed by Spark Therapeutics and licensed by Pfizer in 2014. It was approved by the U.S. last April for the treatment of patients with hemophilia B, with the treatment priced at $3.5 million.

The United States has also approved two other gene therapies for hemophilia: In November 2022, the FDA approved the first gene therapy for hemophilia, Hemgenix (etranacogene dezaparvovec), developed by CSL for the treatment of hemophilia B. In June 2023, the FDA approved the first gene therapy for hemophilia A, Roctavian (valoctocogene roxaparvovec), developed by BioMarin.

Sales of the two drugs have been slow. CSL has not yet disclosed specific figures for Hemgenix, which is priced at $3.5 million in the U.S. market. BioMarin reported Roctavian revenue of $26 million, with the drug priced at $2.9 million. The company has limited sales to the United States, Italy, and Germany, while winding down most of its clinical development efforts to focus on other business areas.

Pfizer's decision to abandon Beqvez essentially marks the company's exit from the AAV gene therapy field. The pharmaceutical company withdrew from early-stage research in 2023, focusing its efforts on late-stage gene therapies for Duchenne muscular dystrophy, hemophilia A, and hemophilia B.

These projects have all been abandoned now. Last July, Pfizer, Inc. abandoned the development of the Duchenne therapy after disappointing Phase III test data, and in the process laid off about 150 people.

Nevertheless, Pfizer appears to remain optimistic about the potential in the hemophilia field but is focusing on non-gene therapy approaches. According to Reuters, Pfizer will continue to invest resources and time into developing Hympavzi, an IgG1 monoclonal antibody that aids in promoting blood clotting. In October 2024, the FDA approved Hympavzi for use in adults and adolescents with Hemophilia A and B, with the drug designed for once-weekly subcutaneous injection.

ComeSource: Rare Disease Information Network