Novo Nordisk’s Alhemo® receives positive opinion from EMA for haemophilia treatment

DENMARK—Novo Nordisk has announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for its haemophilia drug, Alhemo (concizumab).

This decision recommends the approval of Alhemo as the first once-daily subcutaneous prophylactic treatment for individuals aged 12 years or older living with haemophilia A or B with inhibitors.

This development comes just a week after Pfizer, a US pharmaceutical giant, received a green light from the US Food and Drug Administration (FDA) for its haemophilia drug HYMPAVZI (marstacimab-hncq).

HYMPAVZI, like Alhemo, is aimed at providing routine prophylaxis for patients with haemophilia A and B, with the final approval from the European Commission expected within approximately two months.

The CHMP’s positive opinion on Alhemo is based on data from the phase 3 explorer7 study, which evaluated the efficacy and safety of the drug for patients with haemophilia A or B with inhibitors.

If approved by the European Commission, Alhemo will be made available in a pre-mixed and prefilled pen, making it easy to administer subcutaneously.

This delivery method aims to significantly reduce the burden of regular intravenous infusions that many haemophilia patients currently face.

Alhemo® works as an anti-tissue factor pathway inhibitor (TFPI) monoclonal antibody. Its mechanism of action involves blocking a protein called TFPI, which normally prevents blood clotting.

By inhibiting TFPI, Alhemo promotes the production of thrombin, a critical component in the clotting process.

This is especially beneficial for individuals whose clotting factors are either missing or deficient, as it allows blood to clot even in the presence of inhibitors.

Haemophilia is a rare genetic bleeding disorder characterized by the body’s inability to produce sufficient blood clots, which are necessary to stop bleeding.

Traditional treatments for haemophilia involve replacing the missing clotting factors through intravenous infusions, a process known as replacement therapy.

However, some patients develop inhibitors—immune responses that neutralize the effects of the replacement therapy—making treatment much more difficult.

It is estimated that around 30% of individuals with severe haemophilia A develop inhibitors, while approximately 5-10% of those with severe haemophilia B experience the same complication.

Commenting on the CHMP’s positive opinion, Stephanie Seremetis, Novo Nordisk’s Chief Medical Officer for haemophilia, described the decision as a significant milestone.

She emphasized that if approved, Alhemo would become an important addition to the company’s growing haemophilia portfolio, offering daily prophylaxis to prevent bleeds in patients who have developed inhibitors.

Seremetis further highlighted the potential benefits of Alhemo, noting that its prefilled, multi-use pen, which can be stored at room temperature for up to four weeks, could alleviate the physical and emotional burden of treatment.

She pointed out that the simplicity of the subcutaneous administration would give patients greater confidence in managing their daily activities, particularly those living with haemophilia B, who currently have limited treatment options.

With the European Commission’s final decision expected in the coming months, Alhemo could soon provide a new option for haemophilia patients in need of better and more convenient treatments.

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