NICE endorses Sobi's Altuvoct for severe haemophilia A

Sobi's Altuvoct (efanesoctocog alfa), developed in partnership with Sanofi, has received a recommendation from the National Institute for Health and Care Excellence (NICE) for the treatment and prevention of bleeding in haemophilia A patients aged two years and older. In England, approximately 7,700 individuals live with this genetic bleeding disorder, which predominantly affects males. The disorder is characterized by inadequate levels of functional factor VIII, a crucial protein for blood clotting, leading to episodes of bleeding that can result in pain, irreversible joint damage, and potentially life-threatening haemorrhages.

Altuvoct offers a significant advancement in treatment by providing a once-weekly injection designed to substitute the missing factor VIII. This replacement aids in blood clotting, thus temporarily controlling the disorder. Helen Knight, the director of medicines evaluation at NICE, highlighted the challenges associated with current factor VIII replacements, noting the frequent dosing required to avert severe and debilitating bleeding episodes. She emphasized that Altuvoct's weekly administration, combined with its effective bleeding control, could substantially benefit individuals with serious haemophilia A.

The final draft guidance from NICE follows the approval of the therapy by the Medicines and Healthcare products Regulatory Agency in February. This approval was primarily based on the findings from the XTEND-1 study, a late-stage clinical trial involving 159 participants aged 12 years and older with severe haemophilia A. During this year-long study, weekly administration of Altuvoct resulted in 65% of patients experiencing no bleeding episodes, while the remaining 35% reported a significant reduction in bleeding occurrences.

The decision by regulators was further supported by positive outcomes from the XTEND-Kids trial, a phase 3 study involving 74 children under the age of 12 with severe haemophilia A. Additionally, data from a modelling exercise focused on individuals with moderate cases of the disorder contributed to the decision-making process.

James Palmer, NHS England’s medical director for specialised services, described the recommendation as a crucial advancement in treating severe haemophilia A. He emphasized that this development ensures patients continue to have access to the latest advancements in care within the NHS, helping to prevent bleeding episodes.

Sanofi and Sobi's collaboration on efanesoctocog alfa extends beyond the UK, with the therapy marketed as Altuviiio in the US. While Sobi holds the final development and commercialisation rights in Europe, North Africa, Russia, and most Middle Eastern markets, Sanofi retains these rights in North America and other global regions, excluding territories managed by Sobi. This strategic collaboration underscores the commitment of both companies to improve treatment options for haemophilia A patients worldwide, utilizing their combined expertise to advance care and enhance the quality of life for affected individuals.