NICE publishes guidance in England & Wales recommending Altuvoct (efanesoctocog alfa) as a treatment option for people with severe haemophilia A aged 2 years and over

2 April 2025 -- Stockholm, Sweden -- Sobi in the UK today announced that the National Institute for Health and Care Excellence (NICE) has issued its Final Guidance in England and Wales recommending the use of Altuvoct (efanesoctocog alfa). The high-sustained factor VIII replacement therapy will now be available for treating and preventing bleeding episodes in people with severe haemophilia A aged 2 years and over.

The recent Medicines and Healthcare products Regulatory Agency (MHRA) approval noted the reduction in bleeds demonstrated in the trials, highlighting that 65% (86 out of 133) of those in the prophylaxis arm of the XTEND-1 trial had zero bleeds over the course of the year-long study, with a low incidence of bleeds in the remaining 35% (47 out of 133) of people taking part.

Depending on severity of haemophilia, which is defined by factor VIII levels in the blood, patients can experience easy bruising, occasional spontaneous bleeding, and bleeding into joints and muscles.

Frequent bleeding into the joints leads to irreversible haemophilic arthropathy; a debilitating condition associated with inflammation, pain, and joint damage that significantly impacts mobility, everyday activities, and health-related quality of life.[vi]
During the appraisal, the committee heard from patient and clinical experts who advised that this treatment has the potential to improve patient outcomes and provide a meaningful alternative to existing therapies.

The guidance, published earlier today, noted:

• The patient experts involved in the committee hearing stated that their goal is to have a ‘haemophilia free mindset’ but that this is not possible with the current treatment options, in terms of factor VIII replacement therapies. This is due to the frequent dosing schedules and risks of bleeds when on existing EHL and SHL factor VIII replacement therapies
• A new treatment option with effective bleeding control and a less frequent dosing schedule would be welcomed by people with haemophilia A
• Acknowledgment of the impact of the condition and the potential benefits of efanesoctocog alfa as a clinically effective treatment option
• Recognition of efanesoctocog alfa as a cost effective use of NHS resources.

“I am delighted that we now have an additional treatment option for people with severe haemophilia A.” said Dr Pratima Chowdary, Consultant Haematologist and Centre Director at the Katherine Dormandy Haemophilia Centre and an Investigator in the clinical trial. “The clinical trial results show that with weekly treatment factor levels remain within the normal range for more than half the week, while the rest of the time, they are comparable to levels observed in mild haemophilia. Indeed, our experience has shown patients and their families have welcomed this approach due to its simplicity with better control of bleeding, all of which results in improved outcomes for patients.”

Clive Smith, President of the Haemophilia Society, who also has severe haemophilia A, also welcomed the decision. “Altuvoct is a major leap forward in treatment for severe haemophilia A. The increased level of protection it potentially provides, along with less frequent infusions should give people with this condition far more peace of mind and, hopefully, increase their ability to live a full and active life.’

“Despite improvements in haemophilia management over the last 50 years, people living with haemophilia A continue to experience life-threatening bleeds and joint bleeding resulting in pain, loss of function, and impaired work and societal participation, so this positive recommendation from NICE is a significant moment for people living with severe haemophilia A in the UK.” said Sharon Hall, General Manager at Sobi UK and Ireland. “With the potential to significantly improve treatment outcomes and treatment burden we are excited about the positive impact this treatment could have for those living with severe haemophilia A as well as their families and loved ones.”

About Sobi
Sobi is a specialised international biopharmaceutical company transforming the lives of people with rare and debilitating diseases. Providing reliable access to innovative medicines in the areas of haematology, immunology and specialty care, Sobi has approximately 1,800 employees across Europe, North America, the Middle East, Asia and Australia. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. Learn more about Sobi in the UK and the Republic of Ireland at www.sobi.com/uk/en.