AstraZeneca Soliris approved in EU for children, adolescents with refractory generalised myasthenia gravis

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.

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.

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.”

Marc Dunoyer, Chief Executive Officer, Alexion, said, “The impact of gMG on children can be devastating, and families have long been awaiting solutions. This approval of our first-in-class C5 inhibitor Soliris for paediatric patients with refractory gMG in the EU exemplifies our efforts to deliver transformative medicines that help address unmet medical needs for rare disease communities. Soliris offers hope for improved outcomes for children and adolescents impacted by gMG and we are committed to increasing access for these families as quickly as possible.”

The efficacy and safety of Soliris in paediatric patients aged six years and older is consistent with the established profile of Soliris in clinical trials involving adults with refractory gMG. In the Phase III clinical trial of paediatric patients, the majority of reported adverse events were considered mild or moderate.

Soliris was first approved the EU in 2017 for the treatment of certain adults with gMG and is also approved for certain adults with gMG in the US, China and Japan. Regulatory submissions for Soliris for the treatment of paediatric patients with gMG are currently ongoing or planned with multiple health authorities.