The UK Medicines and Healthcare products Regulatory Agency (MHRA) has approved efanesoctocog alfa, marketed as Altuvoct, for the treatment and prevention of bleeding in patients aged 2 years and above with severe or moderate haemophilia A. The marketing authorization was granted on February 14, 2025, to Swedish Orphan Biovitrum AB (Sobi) through an international recognition procedure.
Efanesoctocog alfa functions as a replacement for factor VIII, a protein naturally present in the body that is essential for blood clotting and preventing bleeding. People with severe haemophilia A have undetectable levels of factor VIII and, without treatment, may experience up to 40 bleeding episodes annually.
Julian Beach, MHRA Interim Executive Director of Healthcare Quality and Access, stated: "Patient safety is our top priority, which is why I am pleased to confirm approval of efanesoctocog alfa to treat and prevent bleeding in patients 2 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."
Clinical Evidence Supporting Approval
The approval was supported by robust clinical data. In a study involving 159 patients aged 12 and above with severe haemophilia A, weekly prophylactic injections of Altuvoct resulted in 65% of participants reporting zero bleeding episodes throughout the year-long study period. The remaining 35% experienced significantly reduced bleeding episodes.
Similar efficacy was observed in a separate study of 74 children under 12 years of age with severe haemophilia A. The indication was extended to patients with moderate haemophilia A based on modeling exercises conducted by the company.
The medication is administered intravenously and, like all medicines, may cause side effects, although not all patients experience them. Reported side effects include headaches and arthralgia (joint pain).
NICE Recommendation
The National Institute for Health and Care Excellence (NICE) has also recommended efanesoctocog alfa as a treatment option, with specific conditions. According to NICE guidance, the therapy is recommended for people 2 years and over with haemophilia A only if they have a factor VIII activity level of less than 1% (severe haemophilia A) and the company provides it according to the commercial arrangement.
NICE noted that current treatment options for severe haemophilia A include ongoing treatment with factor VIII replacement therapies (both standard half-life and extended half-life therapies) or emicizumab for prevention, with on-demand factor VIII replacement therapies used to treat bleeding episodes.
While clinical trial results suggest potentially fewer bleeding episodes with efanesoctocog alfa compared to previous factor VIII replacement therapies, NICE acknowledged limitations in the available evidence. Despite uncertainties in the clinical-effectiveness evidence and economic modeling, NICE concluded that efanesoctocog alfa represents a cost-effective use of NHS resources for patients with severe haemophilia A.
Impact for Patients
The approval of efanesoctocog alfa provides an important additional treatment option for patients with haemophilia A in the UK. For individuals with severe haemophilia A, who face significant challenges in managing their condition, this therapy offers potential improvements in bleeding prevention.
The MHRA emphasized that patient safety remains paramount, and as with all products, the safety profile of efanesoctocog alfa will be kept under close review. Patients who suspect they are experiencing side effects are encouraged to discuss this with healthcare professionals and report concerns through the MHRA Yellow Card scheme.
For patients already receiving treatment with efanesoctocog alfa prior to the NICE guidance publication, funding arrangements will remain unchanged until the patient and their healthcare professional consider it appropriate to modify the treatment approach.
