Soliris (eculizumab), the first and only targeted therapy approved for the treatment of paediatric patients with refractory generalised myasthenia gravis (gMG), from Alexion, AstraZeneca Rare Disease has obtained approval for expanded use in the European Union (EU).

The approval allows the treatment of gMG in children and adolescents aged six to 17 years who are anti-acetylcholine receptor (AChR) antibody-positive (Ab+).

The European Commission granted this approval following the positive opinion of the Committee for Medicinal Products for Human Use (CHMP). The decision is backed by results from the Phase III trial of Soliris in paediatric patients with refractory gMG.

The trial confirmed Soliris’ clinical benefit in paediatric patients with refractory gMG who had previously failed immunosuppressive treatment and continued to suffer from significant unresolved disease symptoms. Soliris demonstrated significant improvement in disease severity and function, as measured by the Quantitative Myasthenia Gravis (QMG) total score.

Marc Dunoyer, Chief Executive Officer of Alexion, remarked, “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.”

The efficacy and safety of Soliris in paediatric patients aged six years and older aligns with the established profile of Soliris in clinical trials involving adults with refractory gMG. The most common adverse events in the Phase III clinical trial of paediatric patients were mild or moderate headaches 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.

  Alexion, AstraZeneca, AstraZeneca Rare Disease, CHMP, eculizumab, European Commission, European Union, generalised myasthenia gravis, Marc Dunoyer, SOLIRIS