London – Soliris (eculizumab) has been approved in the European Union (EU) for expanded use to include the treatment of refractory generalised myasthenia gravis (gMG) in children and adolescents aged six to 17 years who are anti-acetylcholine receptor (AChR) antibody-positive (Ab+). This is the first and only targeted therapy approved for the treatment of paediatric patients with the disease in the EU. The approval by the European Commission follows the positive opinion of the Committee for Medicinal Products for Human Use (CHMP) and is based on results from the Phase III trial of Soliris in paediatric patients with refractory gMG.1
In the trial, Soliris demonstrated clinical benefit in paediatric patients with refractory gMG who previously failed immunosuppressive treatment and continued to experience significant unresolved disease symptoms. Soliris showed significant improvement in the primary endpoint of change from baseline in Quantitative Myasthenia Gravis (QMG) total score at week 26, a physician-reported scale assessing disease severity and function (-5.8 [95% CI -8.4, -3.13], p<0.0004). gMG is a rare, debilitating, chronic, autoimmune neuromuscular disease that leads to a loss of muscle function and severe weakness.2
John F. Brandsema, MD, Children’s Hospital of Philadelphia and primary investigator in the Phase III trial of paediatric patients, said: “This approval represents a significant advance in care for paediatric patients with refractory gMG, who previously had no targeted treatment options to help manage their condition. Soliris showed clinical benefit and sustained improvements in disease severity through 26 weeks in a Phase III trial, offering potential to improve quality of life and redefine disease management for children and adolescents living with this rare neurological disease.”