Novo Nordisk to Detail Hemophilia Advancements at ISTH Congress

The upcoming presentations highlight the company’s focus on long-term safety and efficacy across diverse age groups. The FRONTIER4 study results, which include data for both pediatric and adult populations, evaluate the performance of denecimig—a factor VIIIa mimetic—at varying dosing frequencies. These findings arrive as the drug undergoes regulatory review following a Biologics License Application submitted to the U.S. Food and Drug Administration last September.

Beyond the investigational denecimig, the congress will feature the first public look at the explorer10 study. This open-label trial examines the safety and clinical impact of concizumab in children up to 11 years old living with hemophilia A or B who have developed inhibitors. Martin Holst Lange, chief scientific officer at Novo Nordisk, emphasized that the data reflect a commitment to addressing the lived reality of patients, noting that the portfolio expansion targets specific, real-world needs within the rare blood disorders community. The full scope of the company's research includes 18 distinct presentations, ranging from clinical efficacy trials to real-world patient-reported outcomes and pre-clinical studies on thrombin generation.