Altuvoct approved in UK as treatment for hemophilia A patients
Formerly efanesoctocog alfa, therapy is indicated to prevent, treat bleeds
The U.K. Medicines and Healthcare products Regulatory Agency (MHRA) has approved efanesoctocog alfa — to be sold under the brand name Altuvoct — as a treatment for moderate or severe hemophilia A in adults and children, ages 2 and older.
Approved in the European Union in June 2024 under the same brand name, Altuvoct now can also be prescribed in Great Britain to treat and prevent bleeding episodes in hemophilia A patients. The therapy previously was approved in the U.S. in February 2023, where it’s marketed as Altuviiio.
Altuvoct is indicated as a routine prophylactic, or preventive, treatment to lower the frequency of bleeding episodes, as an on-demand treatment to control active bleeds, and for managing bleeds during surgery.
“We’re assured that the appropriate regulatory standards of safety, quality and efficacy for the approval of this new formulation have been met,” Julian Beach, the MHRA’s interim executive director of healthcare quality and access, said in an agency press release.
Approved hemophilia A treatment marketed as Altuviiio in US
The most common form of the rare disease, hemophilia A is caused by mutations in the F8 gene that impair the production or function of a clotting protein called factor VIII, or FVIII. This makes patients susceptible to excessive and prolonged bleeding episodes. The standard of care treatment for hemophilia A is replacement therapy, which involves supplying a functional version of FVIII to restore blood clotting.
Although several replacement therapies are available for hemophilia A, they all generally require frequent dosing, because FVIII is rapidly broken down and doesn’t last long in the bloodstream.
Altuvoct contains a lab-made version of FVIII that is fused to other proteins and protein fragments that help stabilize it, preventing it from being rapidly degraded and prolonging the time it remains in the bloodstream. This allows for less frequent dosing.
Its approval in the U.K. was supported by positive data from the Phase 3 XTEND-1 clinical trial (NCT04161495), which assessed the safety and efficacy of Altuvoct in 159 people, ages 12 and older, with severe hemophilia A.
The treatment was given as an intravenous, or into-the-vein, injection, at a dose of 50 international units per kilogram of body weight. It was administered once weekly to prevent bleeding episodes, or on an on-demand basis to treat bleeding episodes.
Patient safety is our top priority, which is why I am pleased to confirm approval of [Altuvoct] to treat and prevent bleeding in patients 2 years and above with severe or moderate [hemophilia] A. … As with all products, we will keep its safety under close review.
Nearly two-thirds of patients (65%) who received the treatment as prophylaxis had no bleeding episodes over a year of treatment. Altuvoct also improved joint health, eased pain, and effectively treated almost all bleeding episodes when used on-demand.
In the Phase 3 XTEND-Kids trial (NCT04759131), which enrolled 74 children with severe hemophilia A younger than 12, the therapy yielded similar results. None of the participants developed inhibitors, or neutralizing antibodies against the delivered clotting factor that can lower treatment’s efficacy. The therapy’s overall safety profile in younger patients was consistent with that seen in older patients.
“Patient safety is our top priority, which is why I am pleased to confirm approval of [Altuvoct] to treat and prevent bleeding in patients 2 years and above with severe or moderate [hemophilia] A,” Beach said. “As with all products, we will keep its safety under close review.”
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