The authorisation would include certain patients with refractory generalised myasthenia gravis.
UK-based biopharmaceutical company AstraZeneca has announced that the European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) has recommended marketing authorisation for Soliris (eculizumab). The authorisation would include refractory generalised myasthenia gravis (gMG) in children aged six to 17 who are anti-acetylcholine receptor (AChR) antibody-positive (Ab+).
gMC is a rare, debilitating, chronic, autoimmune neuromuscular disorder that leads to a loss of muscle function and severe weakness. Refractory gMG activates the terminal complement cascade ? a part of the body’s immune system ? in an uncontrolled manner, which leads to the terminal cascade over-responding and attacking the body’s own cells. Soliris is a C5 complement inhibitor which works by inhibiting the C5 protein in the terminal cascade.
CHMP’s decision was based on positive results from a phase 3 trial of Soliris in paediatric patients with refractory gMG. Soliris showed significant improvement in the primary endpoint of change from baseline in Quantitiative Myasthenia Gravis (QMG) total score, as well as clinical benefit in patients over six years of age with refractory gMG who had previously unsuccessful immunosuppressive treatment.
John F Brandsema MD, Children’s Hospital of Philadelphia and primary investigator in the phase 3 trial of paediatric patients, said: “Onset of gMG in paediatric patients is extremely rare and complex to manage. As the disease progresses, patients may become nonresponsive to standard treatments, leaving them at risk for serious complications. The phase 3 clinical trial results in patients aged 12 to 17 years with refractory gMG show the efficacy of C5 inhibition in substantially reducing disease severity and symptoms, potentially transforming how this debilitating disease is managed in certain paediatric patients.”
Marc Dunoyer, CEO of Alexion, AstraZeneca’s Rare Diseases division, said: “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 hoping for 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.”