The FDA has announced approval of Soliris injection for treatment of neuromyelitis optica spectrum disorder in adult patients who are anti-aquaporin-4 antibody positive.
The approval was granted to Alexion Pharmaceuticals and is the first for treatment of the rare autoimmune disease.
According to an FDA news release, a study of 143 patients randomized to receive intravenous Soliris (eculizumab) or placebo showed that treatment with the study drug reduced neuromyelitis optica spectrum disorder (NMOSD) relapses by 94% over 48 weeks. Soliris treatment also resulted in fewer hospitalizations and less need for treatment of acute attacks with corticosteroids and plasma exchange.
NMOSD mainly affects the optic nerves and spinal cord. It can be associated with antibodies that bind to aquaporin-4, which can activate other components of the immune system, causing inflammation and damage to the central nervous system. NMOSD typically manifests as clusters of attacks of optic neuritis and transverse myelitis, and follows a course of partial recoveries and remissions. Permanent visual impairment results in about half of patients, according to the release release.
“Soliris provides the first FDA-approved treatment for neuromyelitis optica spectrum disorder, a debilitating disease that profoundly impacts patients’ lives,” said Billy Dunn, MD, director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research. “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.”
Approximately 4,000 to 8,000 patients in the United States are impacted by NMOSD.