FDA Approves Eculizumab for Neuromyelitis Optical Spectrum Disorder

The US Food and Drug Administration (FDA) has granted approval to Alexion Pharmaceuticals for eculizumab (Soliris) for the treatment of neuromyelitis optical spectrum disorder (NMOSD) in adults who are anti-aquaporin-4 (AQP4) antibody positive.

The approval comes on the heels of a 6-month priority review and makes eculizumab the first FDA-approved treatment for NMOSD.

“This approval changes the landscape of therapy for patients with NMOSD,” said Billy Dunn, MD, director of the division of neurology products in the FDA’s Center for Drug Evaluation and Research. “Having an approved therapy for this condition is the culmination of extensive work we have engaged in with drug companies to expedite the development and approval of safe and effective treatments for patients with NMOSD, and we remain committed to these efforts for other rare diseases.”

The approval is based on efficacy demonstrated in a clinical trial of 143 patients who were randomized to receive either intravenous eculizimab injections or placebo over 48 weeks. When comparing treatment with placebo, investigators found that eculizumab reduced the number of NMOSD relapses by 94% over the course of the trial.

Additionally, eculizumab reduced the need for hospitalizations and the need for treatment of acute attacks with corticosteroids and plasma exchange. The most frequently reported adverse reactions reported by patients in the trial were upper respiratory infection, nasopharyngitis, diarrhea, back pain, dizziness, influenza, joint paint, pharyngitis, and contusion.

Eculizumab is only available through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS). Eculizumab has previous FDA approvals dating back to 2007. Eculizumab is approved to reduce the destruction of red blood cells in adults with paroxysmal nocturnal hemoglobinuria, for the treatment of adults with Myastheni Gravis who are anti-acetylcholine receptor antibody positive, and for the treatment of adults and children with atypical hemolytic uremic syndrome.

“Today's approval represents an important milestone for the NMOSD community,” said Victoria Jackson, co-founder of the Guthy-Jackson Charitable Foundation, a non-profit organization dedicated to funding research and raising awareness about NMOSD. “We are thrilled to have partnered with industry to catalyze research and development of targeted therapies to treat NMOSD.”