Pfizer Inc. (NYSE: PFE) has received marketing authorization from the European Commission (EC) for HYMPAVZI™ (marstacimab) for the routine prevention 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%) or severe hemophilia B (congenital factor IX [FIX] deficiency, FIX <1%) without FVIII or 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. It is also the first hemophilia treatment in the EU to be administered via a pre-filled auto-injector pen. The medication offers a subcutaneous treatment option with a once-weekly dosing schedule and minimal preparation required for each administration.
“There is a significant burden associated with the standard treatments for hemophilia A and B, which involve time-consuming preparation and infusions that can lead to missed doses and increased bleeding risks,” said Dr. Laurent Frenzel, Head of the Hemophilia Treatment and Research Center at Necker-Enfants malades Hospital (Paris Cité). “HYMPAVZI represents a significant advancement, offering bleed prevention and the convenience of once-weekly subcutaneous administration via a pre-filled pen.”
Hemophilia, a rare genetic blood disorder caused by a deficiency in clotting factors FVIII (hemophilia A) or FIX (hemophilia B), affects over 800,000 people globally. Diagnosed in early childhood, the disease impairs proper clotting, leading to repeated internal bleeding, particularly in joints, which can result in permanent damage. Despite advancements in treatment, many individuals with hemophilia still experience bleeding episodes and rely on frequent intravenous infusions, often administered multiple times per week.
“HYMPAVZI offers a groundbreaking, first-in-class treatment for people with hemophilia, a disease that frequently causes joint bleeds and impacts daily activities,” said Alexandre de Germay, Chief International Commercial Officer and Executive Vice President, Pfizer. “This approval is a major step forward in Pfizer’s more than four-decade commitment to improving the standard of care in hemophilia. We are excited to offer this medicine, which reduces bleeding episodes compared to factor prophylaxis and requires minimal preparation.”
The EC approval is based on results from the pivotal Phase 3 BASIS study (NCT03938792), which assessed the efficacy and safety of marstacimab in adults and adolescents with severe hemophilia A or B without inhibitors. The study demonstrated that 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. This data showed both non-inferiority and superiority compared to routine prophylaxis with FVIII or FIX. The safety profile was consistent with previous clinical trials, with the most common side effects being injection site reactions, headache, pruritus, and hypertension.
The marketing authorization is valid across all 27 EU member states, as well as in Iceland, Liechtenstein, and Norway. This approval follows the recent regulatory approval of HYMPAVZI in the United States in October.
Pfizer’s ongoing commitment to advancing hemophilia treatment spans over 40 years, beginning with the introduction of recombinant treatments and extending to innovative therapies. Along with the approval of HYMPAVZI, Pfizer has also reported positive Phase 3 results for a gene therapy candidate in hemophilia A (giroctocogene fitelparvovec) and received regulatory approvals for its hemophilia B gene therapy BEQVEZ™ (fidanacogene elaparvovec) in both Europe and the U.S.
