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 positive opinion from the CHMP for Alhemo is a major milestone for patients,” says Stephanie Seremetis, chief medical officer for haemophilia at Novo Nordisk. “If approved, Alhemo would be an important addition to our growing haemophilia portfolio as it offers the potential of everyday prophylaxis to prevent bleeds for people living with haemophilia who have developed the complication of inhibitors. It could alleviate the physical, emotional, and overall treatment burden for people living with haemophilia, as it is delivered in a pre-filled, multi-use, portable pen that can be stored at room temperature for up to four weeks. This could enable patients to have greater confidence in the pursuit of daily activities, which is especially important for people living with haemophilia B with inhibitors, who currently have very limited treatment options.”

Based on data from the phase 3 explorer7 study

The CHMP positive opinion is based on data from the phase 3 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.