Expanded Concizumab Approval Offers Subcutaneous Prophylaxis for Hemophilia Without Inhibitors

The FDA has approved an expanded indication for concizumab-mtci (Alhemo) subcutaneous injection as a once-daily prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A or B without inhibitors.1

The approval follows the original December 2024 approval for adults and children aged at least 12 years with hemophilia A or B with inhibitors.2

"For people living with hemophilia, it is important to continually monitor and discuss bleed control with their healthcare professional," Allison P. Wheeler, MD, Scientific Director, Washington Center for Bleeding Disorders, Seattle, WA, said in a statement.1 "With today's approval of Alhemo® for hemophilia A or B without inhibitors, more people living with these rare blood disorders now have a daily prophylaxis option that may help decrease their bleeding rates."

The approval was based off of data from the phase 3 explorer8 trial, which primarily assessed annual bleeding rate in people receiving concizumab compared to those not using prophylaxis. The end point was met, with a statistically significant reduction in ABR of 79% for patients with hemophilia B (ABR ratio: 0.21; 95% CI: 0.10–0.45; P <.0001) and 86% for patients with hemophilia A (ABR ratio: 0.14; 95% CI: 0.07–0.29; P <.0001).

Furthermore, for patients with hemophilia B, the average ABR decreased to 3.1 (95% CI: 1.9–5.0) and the median ABR to 1.6 (95% CI: 0.0–4.8), compared to baseline values of 14.8 (95% CI: 8.1–26.9) and 14.9 (95% CI: 3.3–22.1), respectively. Among patients with hemophilia A, the average ABR was reduced to 2.7 (95% CI: 1.6–4.6) and the median ABR to 2.9 (95% CI: 0.0–5.2), versus baseline ABRs of 19.3 (95% CI: 11.3–33.0) and 19.6 (95% CI: 17.3–30.4), respectively.

In terms of safety, the most common adverse events reported in at least 5% of patients receiving concizumab were injection site reactions (7%) and headache (7%).

Concizumab is designed to improve thrombin production by blocking TFPI. It is available in prefilled, premixed pens for subcutaneous injection in 60 mg/1.5 mL, 150 mg/1.5 mL, or 300 mg/3 mL formulations.

"The FDA approval of an expanded indication for Alhemo® marks a meaningful step forward for people with hemophilia A or B without inhibitors who are looking for a new prophylaxis treatment option," Anna Windle, PhD, Senior Vice President, Clinical Development, Medical & Regulatory Affairs, Novo Nordisk, added.1 "By building on the initial indication for Alhemo® for those with hemophilia with inhibitors—an especially significant development in hemophilia B where challenges still exist despite advanced treatment options—Novo Nordisk continues its 35+ year legacy in rare bleeding disorders to continue to help address the unmet needs of this community."

REFERENCES

1. FDA approves Alhemo® 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 (HA/HB) without inhibitors. News release. Novo Nordisk. July 31, 2025. https://www.novonordisk-us.com/media/news-archive/news-details.html?id=916409

2. 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. Novo Nordisk. December 20, 2024. Accessed December 23, 2024. https://www.novonordisk-us.com/media/news-archive/news-details.html?id=915084.