On 11 October 2024, Pfizer announced that the US FDA has approved its Hympavzi™ (marstacimab-hncq) for the treatment of adults and adolescents with haemophilia A or B without inhibitors. The approval is based on results from the Phase 3 BASIS trial (NCT03938792) demonstrating substantial bleed reduction compared to routine prophylaxis and on-demand treatment in eligible patients with haemophilia A or B without inhibitors.
Hympavzi™ is the first and only anti-tissue factor pathway inhibitor (anti-TFPI) approved in the US for treating haemophilia A and B, and the first that can be delivered using a pre-filled auto-injector pen.
This news follows the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) adopting a positive opinion for Hympavzi™ for bleeding episodes in patients aged 12 years and older with sever haemophilia A or B in September 2024.