Pfizer swiftly broadens hemophilia B gene therapy with EU approval

Three months after gaining U.S. FDA approval, Pfizer's hemophilia B gene therapy has received a green light from the European Commission to extend its reach within Europe. Known as Beqvez in the United States, the therapy will be marketed under the name Durveqtix in Europe. The European Commission granted a conditional marketing authorization for Durveqtix to be used in adults with severe and moderately severe hemophilia B, specifically those without a history of treatment with factor IX inhibitors or detectable antibodies to variant AAV serotype Rh74.

Durveqtix offers a significant advancement over the current standard of care for hemophilia B, which often requires multiple weekly or biweekly intravenous infusions. Instead, Pfizer’s new therapy enables patients to produce the blood-clotting protein factor IX through a single dose. Alexandre de Germay, Pfizer’s chief international commercial officer, highlighted the substantial medical and treatment burden for people with hemophilia B under current care standards. He pointed out that frequent infusions leave many patients at risk of breakthrough bleeds, causing pain and limiting mobility. Durveqtix has demonstrated potential for long-term bleed protection with just one dose, potentially reducing or eliminating bleeds and transforming hemophilia B care in the European Union.

Pfizer first acquired Durveqtix from Spark Therapeutics in 2014 for an upfront payment of $20 million, with the potential for additional milestone payments totaling $260 million. This acquisition marked Pfizer's entry into the gene therapy market. The European approval follows a recommendation from the European Medicines Agency and marks the therapy’s third approval this year, following Canada in January and the U.S. in April.

However, Pfizer was not the first to market a gene therapy for hemophilia B. CSL and uniQure's Hemgenix received FDA approval in 2022 and made headlines as the world's most expensive drug at the time, priced at $3.5 million. Pfizer matches this price for its own gene therapy in the United States. According to a Pfizer spokesperson, this price point offers a compelling value proposition, potentially delivering savings for the healthcare system by offsetting the long-term costs associated with managing chronic diseases.

Beyond Durveqtix, Pfizer is also working to expand its hemophilia treatment offerings with another gene therapy, giroctocogene fitelparvovec. In a recent phase 3 study, this therapy demonstrated superiority over the standard-of-care treatment for hemophilia A, achieving a statistically significant reduction in the annualized bleed rate compared to baseline characteristics of the patients.

In summary, the approval of Durveqtix by the European Commission represents a significant milestone for Pfizer, enabling the company to offer a transformative treatment option for hemophilia B patients in Europe. By reducing the frequency of treatment and the risk of bleeds, Durveqtix promises to improve the quality of life for those affected by this condition. Pfizer’s continued investment in gene therapy, including its efforts with giroctocogene fitelparvovec, underscores the company's commitment to advancing care for hemophilia patients globally.