Novo Nordisk asks FDA to approve hem A injection therapy Mim8
If approved, it would be sold as a single-use, prefillled disposable pen
Novo Nordisk has submitted an application to the U.S. Food and Drug Administration seeking approval to market Mim8 (denecimig), an injection therapy for preventing bleeds in people with hemophilia A with or without inhibitors.
If approved, Mim8 would be sold as a single-use, prefilled disposable pen for subcutaneous, or under-the-skin, injections administered once every month, once every two weeks, or once every week.
“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,” Anna Windle, senior vice president of clinical development and medical and regulatory affairs at Novo Nordisk, said in a company press release. “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.”
Mim8 designed to mimic the function of factor VIII
Factor replacement therapies are typically the standard treatment for hemophilia A, a genetic disorder caused by a missing or defective factor VIII (FVIII), a protein that helps blood clot. In some patients, the version of FVIII provided by replacement therapies can trigger an immune response and the production of neutralizing antibodies, called inhibitors, which can reduce treatment effectiveness.
Mim8 is an antibody-based therapy designed to bypass the need for FVIII products by mimicking the function of FVIII and stimulating the production of thrombin, an enzyme that helps blood clot. Because Mim8 works without providing an external source of FVIII, it can be used to prevent bleeds in hemophilia A patients with and without inhibitors.
The application requesting its approval was based on positive results from two Phase 3 studies — FRONTIER 2 (NCT05053139) and FRONTIER 3 (NCT05306418) — as well as an open-label extension study called FRONTIER 4 (NCT05685238).
It is important that we continue to drive scientific advances in hemophilia A, and the FRONTIER program supports the potential of denecimig as a [preventive] bleed treatment option for a range of patients across dosing frequencies, age groups, and condition severity, regardless of inhibitor status.
In FRONTIER 2, Mim8 outperformed on-demand or standard preventive therapies in its ability to reduce bleeding episodes in hemophilia A patients ages 12 and older, regardless of inhibitor status. Likewise, FRONTIER 3 showed Mim8 safely and effectively controlled bleeding in children with hemophilia A.
Participants who completed the FRONTIER studies had the option to continue long-term treatment with Mim8 in FRONTIER 4, which is evaluating the efficacy of the treatment given once every two weeks and the long-term safety across all dosing regimens.
“It is important that we continue to drive scientific advances in hemophilia A, and the FRONTIER program supports the potential of denecimig as a [preventive] bleed treatment option for a range of patients across dosing frequencies, age groups, and condition severity, regardless of inhibitor status,” Windle said.
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