The European Commission (EC) has granted marketing authorization for Pfizer’s HYMPAVZI (marstacimab) for the routine prophylaxis of bleeding episodes in patients 12 years of age 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.
HYMPAVZI is 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 the first hemophilia medicine approved in the EU to be administered via a pre-filled, auto-injector pen. HYMPAVZI offers a subcutaneous treatment option with a once-weekly dosing schedule and minimal preparation required for each individual administration.
“There is a considerable treatment burden associated with the standard-of-care options for hemophilia A and B, including time-consuming preparation and administration of infusions and injections potentially causing missed doses and an increased risk of bleeding,” said Dr. Laurent Frenzel, Head of the Hemophilia Treatment and Research Center at the Necker-Enfants malades Hospital (Paris Cité). “HYMPAVZI is a significant advancement for eligible patients in that it may provide bleed prevention as well as once-weekly subcutaneous administration via a pre-filled pen.”
Hemophilia is a family of rare genetic blood diseases caused by a clotting factor deficiency (FVIII in hemophilia A, FIX in hemophilia B), impacting more than 800,000 people globally. Diagnosed in early childhood, hemophilia inhibits the blood’s ability to clot properly, increasing the risk of repeated bleeding inside the joints, which can lead to permanent joint damage. Despite significant progress in hemophilia treatment in recent years, many people living with the disease continue to experience bleeding episodes and manage their condition with frequent intravenous infusions that may need to be administered multiple times a week.
“HYMPAVZI offers a first-in-class treatment option for people living with hemophilia, a disease that often leads to recurring joint bleeds and can impact daily activities as simple as climbing stairs,” said Alexandre de Germay, Chief International Commercial Officer and Executive Vice President, Pfizer. “This approval builds on Pfizer’s more than four-decade commitment to improve the standard of care in hemophilia, and we look forward to delivering this medicine that reduced bleeds as compared to factor prophylaxis and, importantly, requires limited preparation, meeting a key need for eligible patients.”
The marketing authorization is based on results from the pivotal Phase 3 BASIS study that evaluated the efficacy and safety of marstacimab in adults and adolescents 12 years and older with severe hemophilia A or B without inhibitors. In the study, HYMPAVZI 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 active treatment period, demonstrating non-inferiority and superiority compared to routine prophylaxis (RP) with FVIII or FIX administered as part of usual care. The safety profile for HYMPAVZI was consistent with Phase 1/2 results, and the most commonly reported adverse events in the study were injection site reactions, headache, pruritus, and hypertension.
This marketing authorization is valid in all 27 EU member states, as well as in Iceland, Liechtenstein, and Norway. The EC approval follows the regulatory approval of HYMPAVZI in the United States in October.
