Pfizer’s Hympavzi (marstacimab) has been approved by the European Commission (EC) to treat haemophilia A or B in adult and paediatric patients.
The drug has been specifically authorised for the routine prophylaxis of bleeding episodes in patients ages 12 years and older who weigh at least 35kg and have severe haemophilia A without factor VIII inhibitors or severe haemophilia B without factor IX inhibitors.
The EC’s decision makes Hympavzi the first anti-tissue factor pathway inhibitor to be approved in the EU for haemophilia A or B, as well as the first haemophilia medicine approved by the regulator that can be administered via a pre-filled, auto-injector pen.
More than 800,000 people globally are affected by haemophilia, a family of rare genetic blood disorders caused by a clotting factor deficiency.
The disease increases the risk of repeated bleeding inside the joints and, despite recent progress in treatment, many patients continue to experience repeated bleeding episodes and manage their condition with frequent intravenous infusions that may need to be given multiple times a week.
Pfizer’s Hympavzi is given as a once-weekly subcutaneous injection and works by reducing the amount of the naturally occurring anticoagulation protein, tissue factor pathway inhibitor. This increases the amount of thrombin, an enzyme critical in blood clotting, that is generated.
The EU approval, which comes just over one month after the US Food and Drug Administration approved the drug for the same patient population, was supported by positive results from the late-stage BASIS trial.
Hympavzi was shown to reduce the annualised bleeding rate for treated bleeds by 35% during the 12-month active treatment period, demonstrating non-inferiority and superiority compared to routine prophylaxis with factor VIII or factor IX administered as part of usual care.
Alexandre de Germay, chief international commercial officer and executive vice president at Pfizer, said: “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 as simple as climbing stairs… 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.”