SWEDISH ORPHAN BIOVITRUM

Swedish Orphan Biovitrum (Sobi) will present new clinical data at EULAR 2025 in Barcelona, including efficacy and safety outcomes for Gamifant in treating macrophage activation syndrome.

• 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.

• Handok's Empaveli (pegcetacoplan), a C3-targeted therapy, has secured health insurance coverage in Korea for paroxysmal nocturnal hemoglobinuria (PNH) treatment. • Empaveli addresses both intravascular and extravascular hemolysis, offering an alternative for PNH patients who cannot use or have not responded well to C5 inhibitors. • Clinical trials, including PEGASUS and PRINCE, demonstrated Empaveli's superiority over eculizumab in improving hemoglobin levels and reducing the need for transfusions. • Reimbursement criteria prioritize use for patients with contraindications to C5 inhibitors or those with inadequate response or side effects from C5 inhibitor treatments.

• A pilot study suggests that anakinra, an IL-1 antagonist, may reduce dysmenorrhea pain scores in women with endometriosis. • The study also found statistically significant improvements in quality of life, specifically in the domains of powerlessness and self-image. • Anakinra did not disrupt menstrual cycles, suggesting it may preserve fertility potential, a key advantage over current treatments. • Biomarker analysis revealed significantly lower levels of BDNF, an inflammatory marker, indicating a potential anti-inflammatory effect.