The FDA has approved Pfizer's Hympavzi (marstacimab-hncq) for the routine prevention of bleeding episodes in patients 12 years and older with hemophilia A or B.
The approval marks the first non-factor, once-weekly treatment available for hemophilia B without inhibitors, providing a new therapeutic option for those affected by the genetic bleeding disorder.
Hympavzi works by targeting a protein involved in the blood clotting process, rather than replacing the deficient clotting factors VIII or IX typically associated with hemophilia. The treatment reduces the activity of tissue factor pathway inhibitor, a natural anticoagulant protein, increasing the amount of thrombin, a key enzyme in blood clotting.
The FDA’s decision is based on an open-label study involving 116 patients with severe hemophilia A or B. Results showed that patients receiving Hympavzi experienced an annualized bleeding rate of 3.2, compared to 38 during the on-demand factor replacement period.
The nod is also Pfizer’s second win for this indication in 2024. Earlier this year, Pfizer received FDA approval for Beqvez (fidanacogene elaparvovec-dzkt), a one-time gene therapy for adults with moderate to severe hemophilia B. The treatment is approved for patients on routine factor IX (FIX) prophylaxis or those with a history of severe or repeated bleeding episodes, as long as they do not have neutralizing antibodies against the AAVRh74var capsid.
Beqvez is designed to reduce the need for frequent FIX infusions, which are currently required multiple times per week or month. Pfizer has priced Beqvez at $3.5 million per treatment.
