US pharmaceutical major Pfizer Inc. (NYSE: PFE) has announced that the European Commission (EC) has granted approval for its drug Hympavzi (marstacimab-hncq) for the routine prophylaxis of bleeding episodes in patients aged 12 years and older weighing at least 35 kg with severe hemophilia A (congenital factor VIII [FVIII] deficiency, FVIII <1%) without FVIII inhibitors or severe hemophilia B (congenital factor IX [FIX] deficiency, FIX <1%) without FIX inhibitors. This marks the first and only anti-tissue factor pathway inhibitor (anti-TFPI) approved in the European Union (EU) for the treatment of hemophilia A or B, and it is also the first hemophilia medicine approved in the EU to be administered via a pre-filled, auto-injector pen.
Supporting Data from the Phase III BASIS Study
The approval is supported by data from the pivotal Phase III BASIS study, which involved the treatment of 116 eligible severe hemophilia patients with Hympavzi during a 12-month active treatment period (ATP). The study demonstrated that the drug significantly reduced the annualized bleeding rate (ABR) for treated bleeds by 35% (ABR of 5.08 vs. 7.85, p-value 0.0376) during the 12-month ATP. In the 16-month follow-up period of the long-term extension study, Hympavzi consistently reduced the average ABR by 2.79 (95% CI 1.90-4.09), showing non-inferiority compared to routine prophylaxis (RP) in all secondary endpoints related to bleeding, including spontaneous bleeding, joint bleeding, target joint bleeding, and total bleeding.
Hympavzi’s Mechanism of Action and US Fast-Track Status
Hympavzi is a rebalancing agent that targets the Kunitz 2 domain of tissue factor pathway inhibitor (TFPI), making it a potential treatment for both hemophilia A and B patients. The drug was previously awarded fast-track status in the US, highlighting its potential significance in improving the standard of care for hemophilia patients.-Fineline Info & Tech
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