Novo Nordisk denesimig shows long-term safety, efficacy in Hemophilia A study

Novo Nordisk has presented new data from the FRONTIER4 extension study, evaluating the long-term safety and efficacy of its investigational drug denesimig (Mim8) for hemophilia A. The study assessed the drug across once-weekly, once-every-two-weeks, and once-monthly dosing regimens.

The interim analyses included 426 participants of all ages with hemophilia A, with or without inhibitors. The safety profile of denesimig was consistent with previous findings from the FRONTIER research program. Injection-site reactions were reported at low rates and were mild and transient. No evidence of neutralizing antibodies was observed during the study.

Regarding efficacy, the estimated mean annualized bleeding rates (ABRs) remained consistent across all dosing regimens and regardless of inhibitor status. For adults and adolescents, the mean ABR was 0.75, and for children, it was 0.37. Approximately 71% of adults and adolescents and 89% of children experienced zero treated bleeds while on denesimig prophylaxis.

Novo Nordisk submitted a Biologics License Application (BLA) for denesimig to the U.S. Food and Drug Administration (FDA) in September 2025. The company also presented new data from the concizumab explorer10 trial, which demonstrated a significant reduction in bleeding rates for children with hemophilia A or B with inhibitors.

These results reinforce Novo Nordisk's continued efforts to advance treatment options for individuals living with hemophilia.