EU Approves Pfizer’s HYMPAVZI for Hemophilia A and B

Pfizer, a major US pharmaceutical company, has received marketing authorization from the European Commission for its hemophilia treatment, HYMPAVZI (marstacimab). This approval follows positive Phase 3 study outcomes and marks HYMPAVZI as the first once-weekly subcutaneous treatment for severe hemophilia B patients in the EU. It is also the first treatment of its kind that can be administered through a pre-filled auto-injector pen. This European approval came after the FDA’s regulatory approval in October.

Globally, over 800,000 individuals suffer from hemophilia, a rare genetic disorder characterized by a deficiency in clotting factors. Hemophilia A is caused by a lack of the clotting factor FVIII, while hemophilia B is due to a shortage of the clotting factor FIX. People with hemophilia face blood clotting challenges, increasing the risk of internal bleeding in joints and organs especially when injured. Traditionally, managing hemophilia involves regular intravenous infusions of FVIII or FIX, which can be required multiple times a week.

Laurent Frenzel, the Head of the Hemophilia Treatment and Research Center at the Necker-Enfants Malades Hospital in Paris, highlighted the burdens associated with these standard treatments. He explained that the current options involve time-consuming preparation and administration of infusions, leading to missed doses and a heightened risk of bleeding. Frenzel emphasized that HYMPAVZI represents a significant advancement for eligible patients as it potentially offers bleed prevention with a once-weekly subcutaneous administration via a pre-filled pen.

The European Commission's approval was influenced by the positive efficacy and safety outcomes from the pivotal Phase 3 BASIS study. HYMPAVZI was evaluated in adults and adolescents aged 12 or older with severe hemophilia A or B who did not have inhibitors. The study revealed that the drug reduced the frequency of treatments needed for bleeds. Specifically, the annual bleeding rate for treated bleeds saw a 35% reduction. Pfizer noted that these results demonstrated both "non-inferiority and superiority" when compared to routine prophylaxis with FVIII or FIX, which are typically used in standard care.

Alexandre de Germay, Chief International Commercial Officer and Executive Vice President of Pfizer, expressed his views about the European Commission’s decision. He stated that HYMPAVZI provides a first-in-class treatment option for hemophilia patients, a condition that often leads to recurrent joint bleeds and affects daily activities such as climbing stairs. This approval is a testament to Pfizer’s over four-decade-long dedication to improving hemophilia care standards. Germay added that this new treatment reduces bleeding incidents compared to factor prophylaxis and requires minimal preparation, addressing a critical need for eligible patients.