Flexible injection therapy gets EMA panel nod for hemophilia A
CHMP recommends approval of denecimig for adults, children
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A branch of the European Medicines Agency (EMA) has recommended that the experimental therapy denecimig be approved to treat adults and children with hemophilia A, with or without inhibitors.
The recommendation from the EMA’s Committee for Medicinal Products for Human Use (CHMP) will be reviewed by the European Commission, which has the final say over drug approvals in the European Union. The commission isn’t technically required to follow the CHMP’s recommendations, but it almost always does.
Denecimig’s developer, Novo Nordisk, said it expects to secure approval in time to commercially launch the therapy in Europe before the end of 2026. Novo has also applied for U.S. approval of the therapy, which it plans to sell under the brand name Frehemgo.
The company said the drug would be the first prophylactic (preventive) treatment for hemophilia available in a single-use, prefilled pen that could be administered once monthly, weekly, or every other week.
“The combination of strong bleed protection, flexible dosing frequency and a prefilled pen offers a differentiated treatment option that can reduce treatment burden and give people with haemophilia A greater freedom in managing their disease,” Mike Doustdar, president and CEO of Novo, said in a company press release.
‘Important advancement’
“The recommendation by the CHMP for approval of FREHEMGO builds on Novo’s commitment of over 40 years in haemophilia and marks an important advancement for people living with haemophilia A, with or without inhibitors,” Doustdar said.
Hemophilia A is a bleeding disorder marked by reduced activity of the clotting protein factor VIII (FVIII). Standard treatment involves factor replacement therapies, in which a functional version of the FVIII protein is administered to help control bleeding. But some patients develop inhibitors, antibodies against the FVIII protein that reduce the efficacy of replacement therapies.
Denecimig, also known as Mim8, is an antibody-based therapy that’s designed to mimic the activity of the FVIII protein. Since it doesn’t contain the protein itself, the therapy is expected to be unaffected by inhibitors.
Novo’s applications seeking approval of denecimig were based largely on data from two Phase 3 clinical trials, FRONTIER 2 (NCT05053139) and FRONTIER 3 (NCT05306418), as well as a long-term safety and efficacy extension study, FRONTIER4 (NCT05685238). These studies demonstrated that the therapy controlled bleeding in adults, adolescents, and children. Most patients on denecimig have averaged fewer than one bleed per year, according to Novo.
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