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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
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.
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. Denecimig, which is administered under the skin, "mimics" the role of FVIIIa by bridging Factor IXa and Factor X. This action replaces FVIIIa function, which helps restore the body's thrombin generation capacity, helping blood to clot. The use of denecimig in people living with hemophilia A is investigational and not approved by any regulatory authorities worldwide.
Hemophilia is a rare inherited bleeding disorder that impairs the body's ability to make blood clots, a process needed to stop bleeding. 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. There are different types of hemophilia, which are characterized by the type of clotting factor protein that is defective or missing. Hemophilia A is caused by a missing or defective clotting Factor VIII (FVIII). 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. It has been estimated that approximately 30% of people living with hemophilia A have inhibitors.
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.
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.
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