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
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.[iv]
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.[v]
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.i
- A new treatment option with effective bleeding control and a less frequent dosing schedule would be welcomed by people with haemophilia A.i
- Acknowledgment of the impact of the condition and the potential benefits of efanesoctocog alfa as a clinically effective treatment option.i
- Recognition of efanesoctocog alfa as a cost effective use of NHS resources.i
“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 haemophilia
- Haemophilia A is a rare lifelong bleeding disorder that results from the lack, or complete absence of, clotting factor VIII. This means that when people living with haemophilia A bleed, it may take longer to stop.ii
- In someone without haemophilia, their factor VIII levels are usually between 50% and 150%.[vii]
- Severe haemophilia A is defined as a person having a factor VIII activity level of less than 1%.[viii]
- In the UK, there are 9,662 individuals diagnosed with haemophilia A (2023/24 data). Of these, 2,348 patients are registered as having severe disease.iii
About studies
XTEND-1
The clinical evidence for efanesoctocog alfa came from XTEND-1, a phase 3 open-label non-randomised trial with two parallel assignment arms. It evaluated efficacy, safety and pharmacokinetics in 159 previously treated patients (PTPs) 12 years and over with severe haemophilia A and no inhibitors to factor VIII.
Participants in the prophylaxis arm received a weekly prophylactic dose of efanesoctocog alfa for 52 weeks. Participants in on-demand arm received efanesoctocog alfa on demand for 26 weeks followed by a switch to weekly prophylaxis for another 26 weeks.
It found that in a once-weekly fixed prophylactic dose of efanesoctocog alfa 50 IU/kg demonstrated effective bleed protection with a mean annualised bleed rate (ABR) of 0.71.[ix] Superior bleed protection was also demonstrated versus pre-study factor VIII prophylaxis, with a 77% reduction in mean annualised bleeding rate from 2.96 to 0.69 (p<0.001).ix
In terms of its safety profile, the study found that efanesoctocog alfa had an acceptable side effect profile and the development of inhibitors to factor VIII was not detected.ix
XTEND-Kids
XTEND-Kids was an open-label, non-randomised interventional, single-arm study that evaluated efficacy, safety, and pharmacokinetics of efanesoctocog alfa in 74 previously treated patients <12 years of age with severe haemophilia A. Participants received a weekly prophylactic dose of efanesoctocog alfa 50 IU/kg for 52 weeks.
The primary endpoint was the occurrence of inhibitor development, with an overall incidence on trial of 0% (95% confidence interval, 0 to 5). Efanesoctocog alfa also demonstrated effective bleed protection with an estimated mean ABR (95% confidence interval) of 0.89 (0.56-1.42) and a median annualised bleeding rate (ABR) of 0.00 (interquartile range: 0.00-1.02).[x]
In terms of its safety profile, the study found that no serious adverse events were reported, and no factor VIII inhibitors were developed.x
About Altuvoct
Altuvoct is high-sustained factor VIII replacement therapy that contains the active substance efanesoctocog alfa. Studies demonstrated that efanesoctocog alfa had a generally manageable safety profile based on current knowledge.ix,x,[xi] It offers the opportunity of being the first once-weekly factor VIII replacement therapy by maintaining factor VIII levels in the near-normal to normal range (>40%) for approximately 4 days in adultsix and for up to 3 days in children.x Studies found that, at 7 days, factor VIII levels were 15% in adults.ixix For children, the study found factor VIII levels were 10% for 6.7days.x
It is administered once weekly because it has a longer half-life than other factor VIII replacement therapies. It has been shown to provide effective bleed protection, both prophylactically and on-demand, with potential to move towards normal to near-normal factor VIII levels with a single weekly dose.ix,x
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
For more information, please contact
Madeleine Harris Smith, Patient Advocacy and Communications Manager - +44 (0) 7443 191 726 Madeleine.HarrisSmith@sobi.com
[iv] MHRA Recommendation. Efanesoctocog alfa approved to prevent and treat bleeding in children and adults with severe or moderate haemophilia A - GOV.UK [accessed March 2025]
[v] The Haemophilia Society. Symptoms of haemophilia. Symptoms of Haemophilia | The Haemophilia Society [accessed: March 2025]
[vi] Irish Haemophilia Society. Bleeds and Joint Damage Bleeds and Joint Damage - Irish Haemophilia Society [accessed March 2025]
[vii] Genomic Education Programme, Haemophilia A and B - Genomics Education Programme [accessed March 2025]
[viii] The Haemophilia Society How severe is haemophilia? | The Haemophilia Society [accessed March 2025]
[ix] von Drygalski A, Chowdary P, Kulkarni R, Susen S, Konkle BA, Oldenburg J, et al. Efanesoctocog Alfa Prophylaxis for Patients with Severe Hemophilia A. New England Journal of Medicine. 2023;388(4):310-8.
[x] Malec L, Peyvandi, F, Chan CKA. Königs C, et al. Efanesoctocog Alfa Prophylaxis for Children with Severe Hemophilia A. New England Journal of Medicine. 2024;391(3):235-46
[xi] Altuvoct UK, Summary of Product Characteristics ALTUVOCT 1 000 IU powder and solvent for solution for injection - Summary of Product Characteristics (SmPC) - (emc) | 100563 [accessed March 2025]
