EU CHMP Recommends Approval of Soliris for Children and Adolescents with Refractory gMG

Rare Daily Staff

The Committee for Medicinal Products for Human Use of the European Medicines Agency issued a positive recommendation for marketing authorization for AstraZeneca’s Soliris for expanded use to include the treatment of refractory generalized myasthenia gravis in children and adolescents aged 6 to 17 years who are anti-acetylcholine receptor (AChR) antibody-positive (Ab+).

Generalized myasthenia gravis (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 (anti-AChR) that bind to signal receptors at the neuromuscular junction (NMJ), 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 NMJ. 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 developed by the Alexion Rare Diseases division of AstraZeneca. 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.

“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,” said Marc Dunoyer, CEO of Alexion “If approved, Soliris would be the first targeted treatment for pediatric patients living with refractory gMG in Europe, offering the possibility of improved outcomes and quality of life.”

The CHMP based its positive opinion on results from the phase 3 trial of Soliris in pediatric patients with refractory gMG. In the trial, Soliris demonstrated clinical benefit in pediatric 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 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.

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

Photo: Marc Dunoyer, CEO of Alexion