In a letter to the hemophilia community, Pfizer Inc. reported a death due to cerebellar infarction and subsequent cerebral hemorrhage in a long-term extension trial participant taking the New York-based company’s tissue factor pathway inhibitor antagonist Hympavzi (marstacimab).

While recent advances in gene therapy have offered unprecedented options for patients with hemophilia, new data presented at the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, revealed persistent concerns regarding the durability of these treatments and their potential liver toxicity.

While recent advances in gene therapy have offered unprecedented options for patients with hemophilia, new data presented at the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, revealed persistent concerns regarding the durability of these treatments and their potential liver toxicity.

Sanofi SA gained U.S. FDA approval for fitusiran as a first-in-class siRNA therapy for hemophilia. Branded Qfitlia, the antithrombin-lowering therapy is indicated for use as a prophylactic treatment to prevent or reduce bleeding episodes in people with hemophilia A or B, with or without inhibitors.

Sanofi SA gained U.S. FDA approval for fitusiran as a first-in-class siRNA therapy for hemophilia. Branded Qfitlia, the antithrombin-lowering therapy is indicated for use as a prophylactic treatment to prevent or reduce bleeding episodes in people with hemophilia A or B, with or without inhibitors, and joins the small but growing group of non-factor therapy options, with the advantage of a broad label and a convenient dosing regimen.

Researchers at E-Therapeutics plc recently presented efficacy and safety data on ETX-148, a pan-hemophilia agent in murine models of hemophilia A and B.

Sangamo Therapeutics Inc.’s stock sank sharply on the last day of 2025 as Pfizer Inc. handed back the rights to their collaborative gene therapy hemophilia A program. While it was another big loss to Sangamo, which had seen two other major deals fall through in the past two years, the company still has two large collaborations in development.

With Alhemo (concizumab-mtci) from Novo Nordisk A/S, a third hemophilia drug in the past eight months has been approved by the U.S. FDA. The once-daily injectable prophylaxis is to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A or B with inhibitors.