Sobi’s Sanofi-partnered efanesoctocog alfa has been approved by the Medicines and Healthcare products Regulatory Agency (MHRA) to treat haemophilia A.
The drug has been authorised under the brand name Altuvoct to treat and prevent bleeding in patients aged two years and older with severe or moderate cases of the genetic bleeding disorder.
Haemophilia is a rare condition resulting from insufficient levels of functioning factor VIII, a protein that is essential for blood clotting. Patients can experience bleeding episodes that can cause pain, irreversible joint damage and life-threatening haemorrhages.
The active substance in Altuvoct, which is administered as an intravenous injection, replaces the missing factor VIII, helping the blood to clot and providing temporary control of the disorder.
The MHRA’s decision on the drug was based on results from the late-stage XTEND-1 study of 159 patients aged 12 years and older with severe haemophilia A.
Weekly injections of Altuvoct led to 65% of patients reporting zero overall bleeding episodes over the course of the year-long study, while the remaining 35% saw significant reductions in bleeding episodes.
Also supporting the regulator’s decision were positive results from the phase 3 XTEND-Kids trial of 74 children aged under 12 years with severe haemophilia A, as well as data from a modelling exercise of those with moderate cases of the disease.
Julian Beach, MHRA interim executive director of healthcare quality and access, said: “Patient safety is our top priority, which is why I am pleased to confirm approval of [Altuvoct] to treat and prevent bleeding in patients two years and above with severe or moderate haemophilia A.
“We’re assured that the appropriate regulatory standards of safety, quality and efficacy for the approval of this new formulation have been met. As with all products, we will keep its safety under close review.”
Sanofi and Sobi collaborate on the development and commercialisation of efanesoctocog alfa in the US, where it is marketed as Altuviiio, while Sobi has final development and commercialisation rights in Europe, North Africa, Russia and most Middle Eastern markets, with Sanofi holding these rights in North America and all other regions worldwide, excluding the Sobi territory.