First treatment for neuromyelitis optica spectrum disorder receives FDA approval

July 5, 2019

Intravitreal injection treats rare autoimmune disease of the central nervous system

In a press release issued by the FDA, eculizumab (Soliris, Alexion) has been approved by the U.S. Food and Drug Administration to treat neuromyelitis optica spectrum disorder (NMOSD). It is an intravitreal injection for adults who are anti-aquaporin-4 (AQP4) antibody positive. NMOSD is a rare, central nervous system disease which manifests itself in the optic nerves and spinal cord.

“Soliris provides the first FDA-approved treatment for neuromyelitis optica spectrum disorder, a debilitating disease that profoundly impacts patients’ lives,” said Billy Dunn, MD, in a press release. Dr. Dunn is the director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research.

NMOSD causes an autoimmune reaction in which the body attacks optic and spinal cord cells. In cases of the eye, it can cause optic neuritis, a condition that often leads vision pain and loss. According to the release, nearly 50% of patients affected with NMOSD have permanent paralysis and visual loss. 

A clinical study detailing the effectiveness of eculizumab for the treatment of NMOSD was conducted with 143 patients. The patients all had NMOSD with antibodies against AQP4 (anti-AQP4 positive). Patients were randomized into placebo and eculizumab treatment groups. The results showed patients who received eculizumab treatment had a 94% reduction in NMOSD relapse over the trial period, which was 48 weeks.

Read the full announcement here