EU Expands Approval of Soliris to Include Adolescents and Children with Refractory gMG

Rare Daily Staff

The European Union approved expanded use of Alexion, AstraZeneca Rare Disease’s Soliris in children and adolescents aged six to 17 years with the rare autoimmune condition refractory generalized myasthenia gravis who are anti-acetylcholine receptor antibody-positive.

This is the first and only targeted therapy approved for the treatment of pediatric patients with the disease in the European Union. The approval by the European Commission follows the positive opinion of the Committee for Medicinal Products for Human Use and is based on results from the phase 3 trial of Soliris in pediatric patients with refractory generalized myasthenia gravis (gMG).

gMG is a rare autoimmune disorder characterized by loss of muscle function and severe muscle weakness. Eighty percent of people with gMG are AChR antibody positive meaning 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 binding activates the complement system, which is essential to the body’s defense against infection, causing the immune system to attack the neuromuscular junction. This leads to inflammation and a breakdown in communication between the brain and the muscles.

gMG can occur at any age, but it most commonly begins for women before the age of 40 and for men after the age of 60. Initial symptoms may include slurred speech, double vision, droopy eyelids, and lack of balance; these can often lead to more severe symptoms as the disease progresses such as impaired swallowing, choking, extreme fatigue and respiratory failure.

Soliris is a first-in-class C5 complement inhibitor. The medication works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system. When activated in an uncontrolled manner, the terminal complement cascade over-responds, leading the body to attack its own healthy cells. Soliris is administered intravenously every two weeks, following an introductory dosing period.

It was first approved the European Union in 2017 for the treatment of certain adults with gMG and is also approved for certain adults with gMG in the United States, China, and Japan. Regulatory submissions for Soliris for the treatment of pediatric patients with gMG are currently ongoing or planned with multiple health authorities.

Soliris is approved in the United States, European Union, Japan, and China for the treatment of patients with paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome. Soliris is also approved in the European Union for the treatment of certain pediatric patients with gMG, and in the United States, Japan, and China for certain adults with gMG. In addition, Soliris is approved in the United States, European Union, and Japan for the treatment of certain adults with neuromyelitis optica spectrum disorder.

The efficacy and safety of Soliris in pediatric 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 3 clinical trial of pediatric patients, the majority of reported adverse events were considered mild or moderate. The most common adverse events were headache and nasopharyngitis.

In the trial, Soliris demonstrated clinical benefit in pediatric 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.

“The impact of gMG on children can be devastating, and families have long been awaiting solutions,” said Marc Dunoyer, CEO of Alexion. “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.”

Photo: Marc Dunoyer, CEO of Alexion