EC Approves Pfizer's Hympavzi for Haemophilia A and B Treatment

Pfizer’s innovative drug, Hympavzi (marstacimab), has received approval from the European Commission (EC) for use in patients suffering from haemophilia A or B. This approval extends to both adult and pediatric patients, specifically targeting individuals aged 12 and above who weigh at least 35kg. Hympavzi is designed for the routine prophylaxis of bleeding episodes, particularly in cases of severe haemophilia A without factor VIII inhibitors or severe haemophilia B without factor IX inhibitors.

This regulatory approval marks a significant milestone, making Hympavzi the first anti-tissue factor pathway inhibitor to be sanctioned in the European Union for both haemophilia A and B. Additionally, it stands out as the first haemophilia treatment approved in the EU that can be administered using a pre-filled, auto-injector pen, offering a convenient and user-friendly option for patients.

Haemophilia, a rare genetic disorder characterized by a deficiency in blood clotting factors, affects over 800,000 people worldwide. This condition significantly heightens the risk of recurrent bleeding within the joints. Despite advances in medical treatments, many patients still endure frequent bleeding episodes, often managing their condition with regular intravenous infusions that might need to be administered multiple times weekly.

Hympavzi presents a novel approach to treatment, administered as a once-weekly subcutaneous injection. Its mechanism of action involves reducing the levels of the naturally occurring anticoagulation protein, tissue factor pathway inhibitor. By doing so, it increases the production of thrombin, an enzyme essential for blood clotting.

The EC’s decision to approve Hympavzi was supported by positive outcomes from the late-stage BASIS trial. This trial demonstrated that Hympavzi could reduce the annualized bleeding rate for treated bleeding episodes by 35% over a 12-month active treatment period. The drug showed non-inferiority and even superiority compared to standard prophylaxis with factor VIII or factor IX, which are typically used in routine care.

Alexandre de Germay, Pfizer's chief international commercial officer and executive vice president, highlighted the significance of this new treatment option for those living with haemophilia. He noted that Hympavzi offers a first-in-class treatment that addresses the recurring joint bleeds often experienced by these patients, which can impact daily activities, including simple tasks like climbing stairs. Germay also emphasized the reduced bleeding rates achieved with Hympavzi compared to factor prophylaxis and pointed out the drug’s limited preparation requirements, which fulfill a crucial need among eligible patients.

In conclusion, the approval of Hympavzi by the European Commission represents a major advancement in the treatment of haemophilia A and B. It provides a more convenient, efficient, and effective option for the management of this challenging condition, promising improved quality of life for patients by reducing the frequency of bleeding episodes and simplifying the administration process.