Efanesoctocog alfa

Sobi is presenting new clinical outcomes from the XTEND phase 3 program for Altuvoct® (efanesoctocog alfa) at the WFH 2025 Comprehensive Care Summit in Dubai, focusing on joint health and surgical outcomes in hemophilia A patients.

Multiple CDMOs secured significant contract manufacturing opportunities as regulatory bodies approved new indications for established drugs, particularly in oncology and rare diseases.

• Maintaining high Factor VIII (FVIII) levels in people with hemophilia A provides significant clinical benefits, including reduced bleeding risk and better joint health preservation. • Despite advances in prophylactic treatment, standard-of-care prophylaxis does not completely prevent joint damage or chronic pain, with approximately 47% of hemophilia patients experiencing ongoing pain. • Recent innovations in hemophilia treatment, including extended half-life therapies, non-factor replacement options, and gene therapy, offer new approaches to sustaining higher FVIII levels and improving patient outcomes.

• England's NICE has reversed its initial rejection of Altuvoct, Sobi's once-weekly treatment for severe hemophilia A, following the submission of additional evidence. • The positive reimbursement decision marks a significant expansion of treatment options for hemophilia A patients in England, joining approvals in two other European countries. • Altuvoct's approval demonstrates the impact of supplementary evidence in health technology assessment processes, potentially setting a precedent for future drug evaluations.

• NICE has recommended efanesoctocog alfa, a groundbreaking weekly injection therapy, for NHS patients aged two and above with severe haemophilia A, affecting approximately 1,900 people in the UK. • The innovative treatment, priced at £510,000 annually for an average adult, will be available at a confidential discounted rate through an agreement between NHS England and Swedish Orphan Biovitrum. • This once-weekly dosing regimen represents a significant advancement over current factor VIII replacements, offering effective bleeding control while reducing treatment burden for patients with severe haemophilia A.