Source – AstraZeneca
The European Union (EU) has granted approval for the expanded use of Soliris (eculizumab) to treat refractory generalized myasthenia gravis (gMG) in children and adolescents aged six to 17 years who test positive for anti-acetylcholine receptor (AChR) antibodies. This landmark approval makes Soliris the first and only targeted therapy authorized for the treatment of pediatric patients with gMG in the EU.
The European Commission’s decision to approve Soliris followed a positive recommendation from the Committee for Medicinal Products for Human Use (CHMP) and was based on findings from a Phase III clinical trial conducted in pediatric patients with refractory gMG.
During the trial, Soliris demonstrated significant clinical benefits for pediatric patients with refractory gMG who had previously not responded to immunosuppressive treatment and were still experiencing significant unresolved disease symptoms. Notably, Soliris showed remarkable improvement in the primary endpoint of the trial, which assessed disease severity and function using the Quantitative Myasthenia Gravis (QMG) total score. At week 26, the physician-reported scale showed a reduction of -5.8 [95% CI -8.4, -3.13], p<0.0004.
Generalized myasthenia gravis (gMG) is a rare, chronic, and debilitating autoimmune neuromuscular disease that leads to muscle function loss and severe weakness.
“This approval represents a significant advance in care for pediatric 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.”
– John F. Brandsema, MD, Children’s Hospital of Philadelphia and primary investigator in the Phase III trial of pediatric patients
The efficacy and safety of Soliris in pediatric patients aged six years and older align with the established profile of Soliris in clinical trials involving adults with refractory gMG. In the Phase III clinical trial with pediatric patients, the majority of reported adverse events were considered mild or moderate, with headache and nasopharyngitis being the most common.
“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 pediatric 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.”
– Marc Dunoyer, Chief Executive Officer, Alexion
Soliris originally received EU approval in 2017 for treating certain adults with gMG, and it is also approved for certain adults with gMG in the United States, China, and Japan. Currently, regulatory submissions for Soliris to treat pediatric patients with gMG are either underway or planned with several health authorities.
