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Eculizumab granted EU license extension for paediatric refractory generalised myasthenia gravis

2nd August 2023

Eculizumab has been approved by the European Commission for use in those aged six to 17 years who have refractory generalised myasthenia gravis (gMG) and who are also anti-acetylcholine receptor (AChR) antibody-positive (Ab+).

The use of eculizumab (brand name Soliris) has been extended in the European Union (EU) to include the treatment of refractory gMG in children and adolescents, having already been licensed for the treatment of adults with refractory gMG who are AChR antibody-positive. This latest approval represents the first and currently only targeted therapy approved for the treatment of paediatric patients with the disease in the EU.

The extension of eculizumab’s license for use in children and adolescents was based on findings from the Phase III trial of Soliris. Here, eculizumab demonstrated a clinical benefit in paediatric patients with refractory gMG who previously failed immunosuppressive treatment and who continued to experience significant unresolved disease symptoms. 

The drug demonstrated a statistically significant improvement in the primary endpoint, which was a change from baseline in the Quantitative Myasthenia Gravis (QMG) total score – a physician-reported scale assessing disease severity and function – at week 26 (-5.8, 95% CI -8.4, -3.13, p < 0.0004).

Redefining disease management for gMG

Dr John Brandsema, from the Children’s Hospital of Philadelphia and the primary investigator in the study, 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, the manufacturer of Soliris, 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.‘

Generalised myasthenia gravis is a rare autoimmune disorder characterised by loss of muscle function and severe muscle weakness. Some 80% of patients with gMG are AChR antibody positive, which means that they produce specific antibodies that bind to signal receptors at the neuromuscular junction – the connection point between nerve cells and the muscles they control. This effect activates the complement system, causing the immune system to attack the neuromuscular junction and leading to inflammation and a breakdown in communication between the brain and the muscles.

A recent phase 1b/2a study found that using RNA CAR T cell therapy improves clinical symptoms in adult patients with myasthenia gravis.