CHMP Gives Positive Opinion to Novo Nordisk Hemophilia Treatment
As many as 30% of people living with severe hemophilia A develop inhibitors such as TFPI, which can be produced by the body’s immune response to clotting factors in replacement therapy, often limiting the effectiveness of that therapy.
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In a press release on Oct. 18, 2024, Novo Nordisk announced that its Alhemo (concizumab) had been recommended for approval by the European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) (1). If the European Commission (EC) adopts CHMP’s positive opinion, Alhemo would be the first once-daily, subcutaneous, prophylactic treatment for hemophilia A or B with inhibitors in persons 12 years of age or older across the European Union.
“The positive opinion from the CHMP for Alhemo is a major milestone for patients,” said Stephanie Seremetis, chief medical officer for hemophilia at Novo Nordisk, in the press release (1). “If approved, Alhemo would be an important addition to our growing hemophilia portfolio as it offers the potential of everyday prophylaxis to prevent bleeds for people living with hemophilia who have developed the complication of inhibitors. It 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.”
Hemophilia, though rare, is a bleeding disorder in which the body’s ability to make blood clots is impaired. Alhemo is a monoclonal antibody that is designed to block a protein known as TFPI, or tissue factor pathway inhibitor (1). As inhibitors can be produced by the body’s immune response to clotting factors in replacement therapy in hemophilia patients—often given by intravenous infusion—administering concizumab allows for the production of thrombin, which both helps to clot blood and prevent bleeding.
Novo Nordisk said that contemporary estimates suggest that inhibitors are developed by 5– 10% of people living with severe hemophilia B, and as many as 30% of those living with severe hemophilia A (1).
Alhemo was one of 10 medicines recommended by CHMP for approval at its October 2024 meeting, bringing the total number of new medicines that have received positive opinions in 2024 to 89 (2). In September 2024, another hemophilia A and B treatment, Pfizer’s Hympavzi (marstacimab), was also recommended for approval by CHMP (3). FDA approved Hympavzi for use in the United States in October 2024 (4).
Novo Nordisk expects final approval of Alhemo by the EC within two months of CHMP’s October 18 opinion, meaning by mid-to-late December 2024 (1).
References
1. Novo Nordisk. Novo Nordisk A/S: Alhemo Recommended for European Approval as First Once-Daily Subcutaneous Prophylactic Treatment for People Living with Haemophilia A or B with Inhibitors. Press Release. Oct. 18, 2024.
2. EMA. Meeting Highlights from the Committee for Medicinal Products for Human Use (CHMP) 14–17 October 2024. Press Release. Oct. 18, 2024.
3. EMA. Meeting Highlights from the Committee for Medicinal Products for Human Use (CHMP) 16–19 September 2024. Press Release. Sept. 20, 2024.
4. Pfizer. U.S. FDA Approves Pfizer’s HYMPAVZI (marstacimab-hncq) for the Treatment of Adults and Adolescents with Hemophilia A or B Without Inhibitors. Press Release. Oct. 11, 2024.
