Novo Nordisk’s Alhemo Nears EU Approval: A Breakthrough in Haemophilia Treatment

European Approval Recommendation:
The European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of Alhemo (concizumab) as the first once-daily subcutaneous prophylactic treatment for people aged 12 years or older living with haemophilia A or B with inhibitors.

Mechanism of Action:
Alhemo is an anti-tissue factor pathway inhibitor (TFPI) monoclonal antibody that blocks TFPI, ensuring the production of thrombin, which helps to clot the blood and prevent bleeding, even in the presence of inhibitors.

Clinical Trial Results:
The phase 3 explorer7 study showed an 86% reduction in treated spontaneous and traumatic bleeds when on concizumab prophylaxis, with an estimated mean annualised bleeding rate (ABR) of 1.7 compared to 11.8 with no prophylaxis.

Convenience and Accessibility:
Alhemo will be provided in a portable, pre-mixed and prefilled pen, enabling quick and easy subcutaneous administration, which aims to ease the treatment burden of regular intravenous infusions.

Current Approvals:
Alhemo is currently approved in Australia and Switzerland for the treatment of adolescents and adults (12 years or older) with haemophilia A or B with inhibitors. In Japan, it is approved for the treatment of adolescents and adults (12 years or older) with haemophilia A or B with and without inhibitors.

Expected Final Approval:
Novo Nordisk expects a final approval by the European Commission within approximately two months.