Novo Nordisk has announced that it has received a positive opinion from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP), recommending an update of the Alhemo (concizumab) label.

The update would extend its indication to include the treatment of severe haemophilia A and moderate or severe haemophilia B without inhibitors, expanding access beyond its current use in patients with inhibitors.

 “If approved by the European Commission, this label update would extend Alhemo’s convenient once-daily, under-the-skin administration to patients living with haemophilia without inhibitors, providing an efficacious prophylactic choice,” said Martin Holst Lange, executive vice president and head of Development at Novo Nordisk.

“Each person living with haemophilia has individual and evolving needs. With its user-friendly, pre-filled, portable pen, we believe that Alhemo has the potential to offer even more patients an individualised and flexible treatment,” he added.

The company noted that the positive CHMP opinion is based on the results from the phase 3 explorer8 trial, which met its primary endpoint. The study showed that Alhemo prophylaxis led to an 86 percent reduction in treated spontaneous and traumatic bleeds in patients with haemophilia A without inhibitors, and a 79 percent reduction in such bleeds in patients with haemophilia B without inhibitors, compared to no prophylaxis treatment. Alhemo also demonstrated a favourable safety profile in both patient groups.

Additionally, Patient-Reported Outcome (PRO) data from the study indicated improvements in health-related quality of life and reduced treatment burden.

Following the positive opinion from the CHMP, Novo Nordisk expects the European Commission (EC) to approve the label update within approximately two months.

Haemophilia is a rare inherited bleeding disorder that impairs the body’s ability to make blood clots, a process needed to stop bleeding. It is estimated to affect approximately 1,125,000 people worldwide.

Alhemo is an anti-tissue factor pathway inhibitor (TFPI), monoclonal antibody designed to block a protein 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.

Alhemo is currently approved in Europe, the US, India, Brazil, and Switzerland for use in adolescents and adults (12 years or older) with haemophilia A and B with inhibitors. n Japan and Australia, it is approved for patients with and without inhibitors.

In all approved countries, Alhemo is indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes.