Novo Nordisk has secured approval from the US Food and Drug Administration (FDA) for Alhemo (concizumab-mtci) as a once-daily treatment to prevent or reduce bleeding episodes in adults and children aged 12 years and older with haemophilia A or B with inhibitors.
This approval comes after a tumultuous development history for the drug, including a temporary halt of Phase III trials in 2020 due to non-fatal thrombotic events. The trials resumed with enhanced safety protocols, but the FDA initially rejected the drug’s marketing application in 2022, requiring the company to carry out additional data on dosing and monitoring.
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Alhemo has become the first subcutaneous injection treatment available for this patient population, providing an alternative to the intravenous (IV) infusions commonly used for haemophilia treatment. It works by blocking tissue factor pathway inhibitor (TFPI), a protein that limits blood clotting, thereby increasing thrombin production to help manage bleeding episodes in the presence of inhibitors.
The FDA’s decision was supported by data from the Phase III explorer7 study (NCT04083781), which demonstrated an 86% reduction in annual bleeding rates (ABR) among patients treated with Alhemo compared to those without prophylaxis. The study reported a mean ABR of 1.7 for Alhemo-treated patients versus 11.8 for untreated patients.
The approval positions Alhemo as a competitor to Pfizer’s Hympavzi (marstacimab-hncg), another subcutaneous TFPI inhibitor approved in October 2024. However, Hympavzi is administered weekly and is only approved for patients without inhibitors, differentiating it from Alhemo in terms of target population and dosing frequency.
Alhemo is set to generate up to $244m in sales by 2030, whereas Hympavzi is forecast to make $251m in the same year, as per GlobalData’s Pharma Intelligence Center.
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By GlobalDataGlobalData is the parent company of Pharmaceutical Technology.
Novo Nordisk’s announcement comes after the company disclosed disappointing clinical data from its weight-loss therapy CagriSema last week. Despite hitting its primary endpoints, the company’s REDEFINE 1 Phase III trial (NCT05669755) saw patients achieving a weight loss of 22.7% after 68 weeks on CariSema, falling short of the 25% weight loss the company had previously set as its goal.
