FDA

Once-daily hemophilia drug approved for adults, children over 12

December 26, 2024

Brand name: Alhemo

Generic name: concizumab-mtci

Manufacturer: Novo Nordisk

Approval date: December 20, 2024

FDA approved Alhemo (concizumab-mtci), a tissue factor pathway inhibitor (TFPI) antagonist for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients ≥12 years of age with hemophilia A with factor VIII inhibitors or hemophilia B with factor IX inhibitors.

According to a manufacturer press release, this approval marks the first SC injection treatment of its kind for use in this patient population.

Efficacy

Approval was based on data from the multi-national, multi-center, open-label, phase 3 explorer7 trial (NCT04083781) involving 91 adult and 42 adolescent male patients with hemophilia A or B with inhibitors who were prescribed, or were in need of, treatment with therapies that bypass the inhibitor effect.

Efficacy was evaluated by comparing the number of treated bleeding episodes between the Alhemo treatment group and no prophylaxis group. A ratio of the annualized bleeding rates (ABR) was estimated to 0.14, corresponding to a reduction in ABR of 86% for the Alhemo treatment group compared with the no prophylaxis group.

Safety

The most common adverse reactions (reported in ≥5% of patients) in the clinical trial were injection site reactions and urticaria. Treatment with Alhemo may increase the risk of thromboembolic events.

Recommended dose

The recommended dosage of Alhemo consists of a loading dose of 1 mg/kg on day one followed by a once-daily dose of 0.2 mg/kg until individualization of maintenance dose. Four weeks after initiation of treatment, measure concizumab-mtci plasma concentration to individualize the maintenance dose no later than 8 weeks after treatment initiation. See Prescribing Information for details on dosage individualization.

Sources:

FDA. (2024, December 20). FDA approves drug to prevent or reduce the frequency of bleeding episodes for patients with hemophilia A with inhibitors or hemophilia B with inhibitors. [News release]. https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-drug-prevent-or-reduce-frequency-bleeding-episodes-patients-hemophilia-inhibitors-or

Novo Nordisk. (2024, December 20). FDA approves Alhemo injection as once-daily prophylactic treatment to prevent or reduce the frequency of bleeding episodes for adults and children 12 years of age and older with hemophilia A or B with inhibitors. [Press release]. https://www.novonordisk-us.com/media/news-archive/news-details.html

Novo Nordisk: Alhemo (concizumab-mtci). [Package insert]. U.S. Food and Drug Administration website. https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/761315s000lbl.pdf