On 20 November 2024, Pfizer announced that the European Commission (EC) has approved its Hympavzi™ (marstacimab) for the routine prophylaxis of bleeding episodes in patients 12 years of age and older weighing at least 35 kg with severe haemophilia A without FVIII inhibitors or severe haemophilia B without FIX inhibitors. Hympavzi™ is the first and only anti-tissue factor pathway inhibitor (anti-TFPI) approved in the EU for treating haemophilia A and B, and the first that can be delivered using a pre-filled auto-injector pen.
The approval is based on results from the Phase 3 BASIS trial (NCT03938792) which showed that Hympavzi™ significantly reduced the annualised bleeding rate for treated bleeds by 35% during the 12-month active treatment period, demonstrating non-inferiority and superiority compared to routine prophylaxis with FVIII or FIX administered as part of usual care.
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for Hympavzi™ in September 2024. Hympavzi™ was approved by the US FDA in October 2024.