Efanesoctocog alfa (brand name Altuvoct) has been granted a positive opinion by the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) for the treatment and prevention of bleeds and perioperative prophylaxis in haemophilia A, its manufacturer Sobi has announced.
The once-weekly and high-sustained factor VIII replacement therapy is recommended for patients of all ages and any disease severity.
It is the only therapy that has been shown to break through the von Willebrand factor ceiling, which imposes a half-life limitation on current factor VIII therapies, said Sobi.
The European Commission previously granted efanesoctocog alfa Orphan Drug designation in June 2019 and it will now consider a marketing authorisation decision based on the CHMP positive opinion.
Efficacy and safety of efanesoctocog alfa
This recommendation was based on the results from the pivotal phase 3 studies XTEND-1 in adults and adolescents and XTEND-Kids in children, which evaluated the efficacy and safety of efanesoctocog alfa.
XTEND-1 was an open-label, non-randomised interventional study with two parallel assignment arms, which included 159 patients aged 12 years and over with severe haemophilia A. A total of 133 participants in the prophylaxis arm received a weekly prophylactic dose of efanesoctocog alfa for 52 weeks. A further 26 participants in the on-demand arm received efanesoctocog alfa on demand for 26 weeks followed by a switch to weekly prophylaxis for another 26 weeks.
XTEND-Kids was an open-label, non-randomised interventional, single-arm study with 73 patients aged 11 years and younger with severe haemophilia A included in the post hoc analyses. Participants received a weekly prophylactic dose of efanesoctocog alfa for 52 weeks.
These studies demonstrated that once-weekly efanesoctocog alfa prophylaxis (50 IU/kg) provided significant bleed protection (mean ABR <1 and 80-88% of patients free from spontaneous bleeds) in people with severe haemophilia A of any age.
The data also showed substantial improvement in joint health, physical health, pain and overall quality of life when comparing Week 52 and baseline measurements.
The researchers concluded that efanesoctocog alfa significantly improved protection from bleeds compared to existing factor VIII prophylaxis.
Lydia Abad-Franch, head of research, development, and medical affairs, and chief medical officer at Sobi , said: ‘Efanesoctocog alfa sustains high factor VIII activity levels throughout the week, giving patients confidence in the protection it can provide to prevent bleeds, manage surgery, and resolve bleeds.
‘With the potential to significantly improve treatment outcomes and quality of life for people living with haemophilia A, we are excited about the positive impact this treatment could have around the world.’
