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The European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of Novo Nordisk’s brand of concizumab (Alhemo) for treatment of hemophilia A or B in patients over 12 years of age.

Concizumab is a monoclonal antibody that acts by blocking the tissue factor pathway inhibitor, thereby allowing the production of thrombin, an endogenous protein that aids in clot formation and prevents bleeding.

Patients primarily use it to prevent or reduce the frequency of bleeding episodes.

If approved, Alhemo will be the first once-daily prophylaxis medication administered subcutaneously in the management of patients with hemophilia A or B with inhibitors.

Standard treatment in hemophilia management involves intravenous replacement therapy with the missing clotting factors.

However, a physiological immune response can trigger the production of clotting factor inhibitors, rendering this treatment strategy ineffective and thereby narrowing therapeutic options for patients.

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The recommendation by the CHMP is based on results from a study that showed that there was an 86% reduction in treated spontaneous and traumatic bleeds among patients administered with concizumab prophylaxis, having an estimated mean annualized bleeding rate of 1.7 compared to 11.8 with no prophylaxis.

Due to its unique mechanism of action, Alhemo ensures clotting happens despite the presence of inhibitors.

Stephanie Seremetis, Chief Medical Officer for hemophilia at Novo Nordisk, has emphasized that CHMP’s recommendation is a “major milestone” for hemophilia patients.

“Alhemo could alleviate the physical, emotional, and overall treatment burden for people living with hemophilia, 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 hemophilia B with inhibitors, who currently have very limited treatment options," added Stephanie.

Currently, Alhemo is authorized for hemophilia management in the Japan, Canadian, and Swiss markets.

It is yet to gain approval in the US, with the FDA seeking clarification on the applied monitoring and dosing of patients as well as the drug's manufacturing process.

Within approximately two months, Novo Nordisk expects a final approval by the European Commission.

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