Soliris approved in Japan for paediatric patients with generalised myasthenia gravis (gMG)

Soliris is the first and only targeted therapy approved for the treatment of children and adolescents with gMG in Japan.

The approval by the Japanese Ministry of Health, Labour and Welfare (MHLW) was based on results from the Phase III trial of Soliris in paediatric patients with refractory gMG. 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.

Keiko Ishigaki, MD, PhD, Department of Pediatrics, Tokyo Women's Medical University, School of Medicine, Tokyo, Japan, said: “gMG is challenging to manage in paediatric patients, as current therapies available to this population, such as immunosuppressants, may not offer adequate control as the disease progresses. Today’s expanded approval of Soliris in Japan demonstrates the impact of C5 complement inhibition in treating gMG, offering paediatric patients a targeted option with the potential to preserve muscle function and reduce disease severity.