Alexion receives positive CHMP opinion for Soliris for the treatment of neuromyelitis optica spectrum disorder

Alexion Pharmaceuticals, Inc. announced that the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion to extend the current marketing authorization of Soliris (eculizumab) to include the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive with a relapsing course of the disease. The European Commission will review the CHMP opinion and the final decision is anticipated within two months.

NMOSD is a rare, devastating, complement-mediated disorder of the central nervous system characterized by relapses, also referred to as attacks. Each attack results in stepwise accumulation of disability, including blindness and paralysis and sometimes premature death. NMOSD disproportionately strikes young women in the prime of their lives, with the average age of first onset at just 39 years. Previously known as Devic’s Disease, NMOSD is often confused with other neurological illnesses such as multiple sclerosis (MS), which can lead to delays in diagnosis and treatment with medicines that can worsen disease progression.

Comment: The positive opinion is based on comprehensive results from the Phase III randomized, double-blind placebo controlled PREVENT trial, which were published in The New England Journal of Medicine and a long-term extension study (ECU-NMO-302), which is still underway.