Sobi’s Sanofi-partnered Altuvoct (efanesoctocog alfa) has been recommended by the National Institute for Health and Care Excellence (NICE) to treat and prevent bleeding in haemophilia A patients aged two years and older.
Approximately 7,700 people in England are living with the genetic bleeding disorder and males are disproportionately affected.
The condition results from insufficient levels of functioning factor VIII, a protein that is essential for blood clotting, and patients can experience bleeding episodes that can cause pain, irreversible joint damage and life-threatening haemorrhages.
Altuvoct, which can be given as a once-weekly injection, is designed to replace the missing factor VIII, helping the blood to clot and providing temporary control of the disorder.
“Current factor VIII replacements can be difficult to manage due to the need for frequent dosing to prevent potentially life-threatening and debilitating bleeding episodes,” explained Helen Knight, director of medicines evaluation at NICE. “[Altuvoct] only has to be taken once-a-week. Combined with its effective bleeding control, it has the potential to have a significant positive impact for some people with severe haemophilia A.”
NICE’s final draft guidance follows the Medicines and Healthcare products Regulatory Agency’s approval of the therapy in February, which was based on results from the late-stage XTEND-1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 cases of the disorder, as well as data from a modelling exercise of those with moderate cases.
James Palmer, NHS England’s medical director for specialised services, said: “This is an important step forward in treatment for patients with severe haemophilia A and ensures they continue to have access to the latest advances in care on the NHS to help prevent bleeding episodes.”
Sanofi and Sobi collaborate on 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.