Soliris has been recommended for marketing authorisation 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+).
If authorised, Soliris would be the first and only targeted therapy approved for the treatment of paediatric patients aged six years and older with refractory gMG in the EU.”
“gMG can impact patients’ ability to walk, talk, breathe and participate in routine activities, and with no existing targeted therapies for children and adolescents with this condition, families have long been hopeful for a treatment. If approved, Soliris would be the first targeted treatment for paediatric patients living with refractory gMG in Europe, offering the possibility of improved outcomes and quality of life,” says Marc Dunoyer, Chief Executive Officer, Alexion.
Based its opinion on results from Phase III trial
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) based its positive opinion 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 aged six years and older 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.
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. The most common adverse events were headache and nasopharyngitis.
Soliris was first approved in 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.
Photo of Marc Dunoyer: AstraZeneca