CHMP positive for 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

Haemophilia is a rare bleeding disorder that impairs the body’s ability to make blood clots, a process needed to stop bleeding. Haemophilia is often treated by replacing the missing clotting factor via intravenous infusions, also known as replacement therapy. However, sometimes the body can produce inhibitors as an immune response to the clotting factors in the therapy, which means replacement therapy does not work and limits treatment options overall. Currently, it is estimated that up to 30% of people living with severe haemophilia A develop inhibitors, and 5-10% of those with severe haemophilia B develop inhibitors.

Alhemo is an anti-tissue factor pathway inhibitor (TFPI) monoclonal antibody that, if approved by the European Commission, will offer the first once-daily subcutaneous prophylactic treatment to people with haemophilia A or B with inhibitors. Alhemo is designed to block a protein called TFPI in the body that stops blood from clotting. By blocking TFPI, Alhemo ensures the production of thrombin, which helps to clot the blood and prevent bleeding, even when the other clotting factors are missing or deficient. Importantly, this means Alhemo  allows blood clots to happen, even in the presence of inhibitors.

The CHMP positive opinion is based on data from the phase III explorer7 study, which was designed to evaluate the efficacy and safety profile of Alhemo for people living with haemophilia A or B with inhibitors. If approved, Alhemo will be provided in a portable, pre-mixed and prefilled pen, enabling quick and easy subcutaneous administration with the aim of easing the treatment burden of regular intravenous infusions. Novo Nordisk expects a final approval by the European Commission within approximately two months.