Novo Nordisk’s Alhemo® Nears Approval as First Daily Treatment for Haemophilia with Inhibitors

Novo Nordisk has announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion, recommending the approval of Alhemo® (concizumab). If approved, Alhemo® would become the first once-daily subcutaneous prophylactic treatment for individuals aged 12 and older with haemophilia A or B who have developed inhibitors.

Haemophilia is a rare condition that impairs the body's ability to form blood clots, which are essential for stopping bleeding. The standard treatment involves intravenous infusions of the missing clotting factors, but some individuals develop inhibitors, making this therapy ineffective. Around 30% of people with severe haemophilia A and 5-10% of those with severe haemophilia B develop these inhibitors, limiting their treatment options.

Alhemo® is a monoclonal antibody that targets tissue factor pathway inhibitor (TFPI), a protein that prevents blood clotting. By blocking TFPI, Alhemo® promotes the production of thrombin, enabling clot formation even in the absence of certain clotting factors, making it effective despite the presence of inhibitors.

Stephanie Seremetis, chief medical officer for haemophilia at Novo Nordisk, emphasized the significance of the CHMP's positive opinion, noting that Alhemo® could be a valuable addition to their haemophilia portfolio. If approved, it would offer everyday prophylaxis to prevent bleeding episodes and could reduce the physical and emotional burden of treatment, particularly for those with haemophilia B and inhibitors, who currently have limited treatment options. Alhemo® is administered through a pre-filled, multi-use portable pen that can be stored at room temperature for up to four weeks, potentially offering patients more confidence in their daily activities.

The CHMP’s recommendation is based on results from the phase 3 explorer7 study, which evaluated the efficacy and safety of Alhemo® in patients with haemophilia A or B with inhibitors. If approved, Alhemo® will be available in a portable, pre-mixed pen for easy subcutaneous administration, simplifying treatment compared to regular intravenous infusions. Novo Nordisk anticipates final approval from the European Commission within the next two months.