Novo Nordisk submits Biologics License Application (BLA) to FDA for Mim8; an investigational, prophylaxis treatment for people living with hemophilia A with or without inhibitors

• If approved, Mim8 (denecimig) would offer the flexibility of treatment with a single-use, prefilled disposable pen injector with dosing options including once every month, once every two weeks, and once every week for people living with hemophilia A
  
  
• In hemophilia A, denecimig is designed to help the body form blood clots more effectively and was studied as part of the FRONTIER program across different dosing frequencies, age groups, severities, and with or without inhibitors¹
  
  
• Novo Nordisk is advancing its long-standing commitment to people living with hemophilia A, a potentially life-threatening condition

BAGSVÆRD, Denmark and PLAINSBORO, N.J., Sept. 29, 2025 /PRNewswire/ -- Today, Novo Nordisk announced Mim8 (denecimig) has been submitted for review to the US Food and Drug Administration (FDA) through a Biologics License Application (BLA), a formal request to evaluate a biologic medicine. Denecimig is an investigational, next-generation bispecific antibody Factor VIIIa (FVIIIa) mimetic, designed as a preventive bleed routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients with hemophilia A (congenital FVIIIa deficiency), with or without inhibitors.² If approved, denecimig will be the first FVIIIa mimetic with flexible once every month, once every two weeks, and once every week dosing in a pre-filled, single-use pen, catering to the diverse needs of people living with hemophilia A, with or without inhibitors.¹

"It is important that we continue to drive scientific advances in hemophilia A, and the FRONTIER program supports the potential of denecimig as a prophylaxis bleed treatment option for a range of patients across dosing frequencies, age groups, and condition severity, regardless of inhibitor status," said Anna Windle, Senior Vice President, Clinical Development, Medical & Regulatory Affairs at Novo Nordisk. "A cornerstone of hemophilia treatment is to help prevent bleeding episodes, and this submission is another step forward in our long-standing mission to help improve care. If approved, denecimig's single-dose prefilled injector pen and flexible dosing schedule may better fit into the lives of people living with this challenging condition."

The FDA submission is grounded in the results from the FRONTIER study program, a comprehensive program of studies designed to establish the efficacy and safety profile of denecimig as a prophylaxis treatment to prevent or reduce the frequency of bleeding episodes in people with hemophilia A, with or without inhibitors.^1,3-6

About Mim8 (denecimig)
Mim8 (denecimig) is an investigational FVIIIa mimetic bispecific antibody designed with the aim to deliver once every month, once every two weeks, or once every week prophylaxis for people living with hemophilia A, with or without inhibitors.^3-6 Denecimig, which is administered under the skin, "mimics" the role of FVIIIa by bridging Factor IXa and Factor X.⁷ This action replaces FVIIIa function, which helps restore the body's thrombin generation capacity, helping blood to clot.⁸ The use of denecimig in people living with hemophilia A is investigational and not approved by any regulatory authorities worldwide. 

About Hemophilia A
Hemophilia is a rare inherited bleeding disorder that impairs the body's ability to make blood clots, a process needed to stop bleeding.⁹ According to the World Federation of Hemophilia, it is estimated to affect approximately 836,000 people worldwide and hemophilia A is estimated to account for 80-85% of all hemophilia cases.¹⁰ There are different types of hemophilia, which are characterized by the type of clotting factor protein that is defective or missing.⁶ Hemophilia A is caused by a missing or defective clotting Factor VIII (FVIII).⁹ Some people with hemophilia A may develop inhibitors, an immune system response to the clotting factors used in replacement therapy, which can cause treatment to become ineffective.¹¹ It has been estimated that approximately 30% of people living with hemophilia A have inhibitors.¹¹

About the FRONTIER Clinical Program 
The FRONTIER clinical program includes FRONTIER1-5 and investigates denecimig as a preventive bleed treatment across pediatric and adult populations with hemophilia A, with or without inhibitors. ^1,3-5,12,13

FRONTIER2, FRONTIER3 and FRONTIER4 formed the basis of the denecimig BLA submission. FRONTIER2 evaluated denecimig treatment once every month and once every week in adults and adolescents 12 years of age and older; FRONTIER3 evaluated denecimig treatment once every month and once every week in children below the age of 12; and FRONTIER4 was an open label extension trial evaluating the efficacy of denecimig once every two weeks (Q2W) as well as investigating the long-term safety of denecimig across all dosing regimens (once every month, once every two weeks, and once every week) in subjects with hemophilia A, with or without inhibitors.^3-5

Novo Nordisk is a leading global healthcare company founded in 1923 and headquartered in Denmark. Our purpose is to drive change to defeat serious chronic diseases built upon our heritage in diabetes. We do so by pioneering scientific breakthroughs, expanding access to our medicines, and working to prevent and ultimately cure disease. Novo Nordisk employs about 78,400 people in 80 countries and markets its products in around 170 countries. For more information, visit  novonordisk.com, Facebook, Instagram, X, LinkedIn, and YouTube.  

Contacts for further information

References

1. Novo Nordisk Data on File.
2. Bowyer AE, Hickey K, Kitchen S, Ezban M. A next generation FVIII mimetic bispecific antibody, Mim8, the impact on non-factor VIII related haemostasis assays. Haemophilia. 2023;29(6):1633-1637.
3. ClinicalTrials.gov. A Research Study Investigating Mim8 in Adults and Adolescents With Haemophilia A With or Without Inhibitors. Last accessed September 2025. Available at https://clinicaltrials.gov/study/NCT05053139.
4. ClinicalTrials.gov. A Research Study Looking at Mim8 in Children With Haemophilia A With or Without Inhibitors. Last accessed September 2025. Available at https://clinicaltrials.gov/study/NCT05306418.
5. ClinicalTrials.gov. A Research Study Looking at Long-term Treatment With Mim8 in People With Haemophilia A (FRONTIER 4). Last accessed September 2025. Available at https://clinicaltrials.gov/study/NCT05685238.
6. Østergaard H, Lund J, Greisen PJ, et al. A factor VIIIa-mimetic bispecific antibody, Mim8, ameliorates bleeding upon severe vascular challenge in hemophilia A mice. Blood. 2021;138(14):1258-1268.
7. Lentz S, Chowedary P, Gil L, et al. FRONTIER1: a partially randomized phase 2 study assessing the safety, pharmacokinetics, and pharmacodynamics of Mim8, a factor VIIIa mimetic. J Thromb Haemost. 2024;22(4): 990-1000.
8. U.S. National Library of Medicine. F8 gene. MedlinePlus Genetics. Last accessed August 2025. Available at https://medlineplus.gov/genetics/gene/f8/.
9. MedlinePlus. Hemophilia. Last accessed September 2025. Available at https://medlineplus.gov/genetics/condition/hemophilia.
10. World Federation of Hemophilia. World Federation of Hemophilia Report on the Annual Global Survey 2023. Last accessed September 2025. Available at https://wfh.org/research-and-data-collection/annual-global-survey/.
11. Kim JY, You CW. The prevalence and risk factors of inhibitor development of FVIII in previously treated patients with hemophilia A. Blood Res. 2019;54:204-209.  
12. ClinicalTrials.gov. A Research Study Investigating Mim8 in People With Haemophilia A (FRONTIER1). Last accessed September 2025. Available at https://clinicaltrials.gov/study/NCT04204408.
13. ClinicalTrials.gov. A Research Study Looking at How Safe it is to Switch From Emicizumab to Mim8 in People With Haemophilia A (FRONTIER 5). Last accessed September 2025. Available at https://clinicaltrials.gov/study/NCT05878938.

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