European Commission Approves Pfizer's Hympavzi for Haemophilia A and B
Key Insights
The European Commission (EC) has approved Pfizer's Hympavzi (marstacimab) for routine prophylaxis of bleeding episodes in haemophilia A (search) and B patients.
Hympavzi is the first anti-tissue factor pathway inhibitor (search) approved in the EU for haemophilia A (search) or B, administered via a pre-filled, auto-injector pen.
The approval was supported by the late-stage BASIS trial, which demonstrated a 35% reduction in annualized bleeding rate compared to factor VIII or IX prophylaxis.
The European Commission (EC) has granted approval to Pfizer’s Hympavzi (marstacimab) for the treatment of haemophilia A (search) or B in both adult and paediatric patients. Specifically, the drug is authorized for routine prophylaxis to prevent bleeding episodes in patients 12 years and older, weighing at least 35kg, who have severe haemophilia A without factor VIII inhibitors or severe haemophilia B (search) without factor IX inhibitors. This decision marks a significant advancement in the treatment of haemophilia, offering a novel approach to managing the condition.
Hympavzi stands out as the first anti-tissue factor pathway inhibitor (search) to receive approval in the EU for haemophilia A (search) or B. It is also the first haemophilia medicine approved by the regulator that offers the convenience of a pre-filled, auto-injector pen for administration. Haemophilia, a rare genetic blood disorder affecting over 800,000 people globally, results from a deficiency in clotting factors, leading to an increased risk of internal bleeding, particularly in the joints. Despite advancements in treatment, many patients still experience recurrent bleeding episodes and rely on frequent intravenous infusions, sometimes multiple times per week.
Hympavzi is administered as a once-weekly subcutaneous injection. It functions by reducing the levels of tissue factor pathway inhibitor (search), a naturally occurring anticoagulation protein. This reduction subsequently increases the amount of thrombin, a crucial enzyme in the blood clotting process, thereby promoting effective clot formation.
The EU approval was supported by data from the late-stage BASIS trial, aligning with the US Food and Drug Administration's (FDA) approval one month prior for the same patient population. The BASIS trial demonstrated that Hympavzi reduced the annualized bleeding rate for treated bleeds by 35% during the 12-month active treatment period. This result confirmed non-inferiority and superiority compared to routine prophylaxis with factor VIII or factor IX, which are administered as part of the standard care regimen.
Alexandre de Germay, chief international commercial officer and executive vice president at Pfizer, stated that Hympavzi offers a first-in-class treatment option for people living with haemophilia, a disease that often leads to recurring joint bleeds and can impact daily activities. He added that they 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.