Soliris Gains FDA Approval for Neuromyelitis Optica Spectrum Disorder

The FDA Thursday approved Alexion Pharmaceuticals’ brand-name eculizumab (Soliris) for intravenous use for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive.

It is the first FDA-approved treatment for the autoimmune disease and the fourth indication for Soliris, which carries a list price of approximately $500,000 per year.

NMOSD mainly affects the optic nerves and spinal cord. NMOSD can be associated with antibodies that bind to the AQP4 protein; this binding appears to activate other components of the immune system, causing inflammation and damage to the central nervous system. NMOSD is believed to affect 4000 to 8000 patients in the United States.

The effectiveness of Soliris for the treatment of NMOSD was demonstrated in a clinical study of 143 patients with NMOSD who had AQP4 antibodies who were randomized to receive either Soliris or placebo. Compared with treatment with placebo, the study showed that treatment with Soliris reduced the number of NMOSD relapses by 94% over the 48-week course of the trial. Soliris also reduced the need for hospitalizations and the need for treatment of acute attacks with corticosteroids and plasma exchange.

“Soliris provides the first FDA-approved treatment for neuromyelitis optica spectrum disorder, a debilitating disease that profoundly impacts patients’ lives,” Billy Dunn, MD, director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research, said in a statement. “This approval changes the landscape of therapy for patients with NMOSD. 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.”

In NMOSD, the body's immune system mistakenly attacks healthy cells and proteins. Individuals with NMOSD typically have attacks of optic neuritis, which causes eye pain and vision loss. Individuals also can have attacks resulting in transverse myelitis, which often causes numbness, weakness, or paralysis of the arms and legs, along with loss of bladder and bowel control. Most attacks occur in clusters, days to months to years apart, followed by partial recovery during periods of remission. Approximately 50% of patients with NMOSD have permanent visual impairment and paralysis caused by NMOSD attacks.

According to the National Institutes of Health, women are more often affected by NMOSD than men; African Americans are at greater risk of the disease than Caucasians.

Soliris, first approved in 2007, is also approved to treat a rare blood disease called paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome to inhibit complement-mediated thrombotic microangiopathy, and adults with myasthenia gravis.