HYMPAVZI™ Wins EU Approval For Treating Hemophilia A And B

Pfizer Inc. has secured European Commission (EC) approval for HYMPAVZI™ (marstacimab), a groundbreaking treatment for managing severe hemophilia A or B in patients aged 12 and older who weigh at least 35 kg and lack factor VIII (FVIII) or factor IX (FIX) inhibitors. HYMPAVZI™ is the first anti-tissue factor pathway inhibitor (anti-TFPI) authorized in the European Union (EU) for hemophilia A or B and stands out as the first EU-approved hemophilia therapy delivered via a pre-filled, auto-injector pen. This innovative option enables patients to manage their condition with a simple, once-weekly subcutaneous injection, requiring minimal preparation for each dose.

Hemophilia is a rare inherited disorder affecting blood clotting due to a deficiency in clotting factors—factor VIII in hemophilia A or factor IX in hemophilia B. Affecting over 800,000 people worldwide, this condition is often identified in early childhood. It disrupts normal blood clotting, leading to frequent joint bleeding, which can cause long-term joint damage. While advancements in hemophilia treatment have been made, many patients still face challenges such as recurring bleeding episodes and the need for frequent intravenous infusions, sometimes multiple times a week, to manage their condition.

Dr. Laurent Frenzel, Head of the Hemophilia Treatment and Research Center at the Necker-Enfants malades Hospital (Paris Cité), said in a statement, “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. 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.”

Alexandre de Germay, Chief International Commercial Officer and Executive Vice President, Pfizer, also stated, “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. 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 European Commission’s approval of HYMPAVZI™ is supported by data from the pivotal Phase 3 BASIS study, which evaluated its effectiveness and safety in patients aged 12 and older with severe hemophilia A or B without inhibitors. The study demonstrated a 35% reduction in the annualized bleeding rate (ABR) for treated bleeds (ABR of 5.08 vs. 7.85, p=0.0376) over a 12-month treatment period. HYMPAVZI not only proved non-inferior but also superior to routine prophylaxis (RP) with FVIII or FIX. Its safety profile aligned with earlier trials, with the most common side effects including injection site reactions, headaches, itching, and hypertension.  

This approval applies across all 27 EU countries, as well as Iceland, Liechtenstein, and Norway. It follows HYMPAVZI’s regulatory approval in the United States in October. Pfizer’s commitment to advancing hemophilia treatment spans over four decades, starting with the introduction of recombinant therapies and evolving into cutting-edge innovations. Alongside HYMPAVZI, Pfizer has made strides with its hemophilia A gene therapy candidate, giroctocogene fitelparvovec, which showed positive Phase 3 results earlier this year, and its hemophilia B gene therapy, BEQVEZ™ (fidanacogene elaparvovec), recently approved in Europe and the U.S.