FDA Approves Uplizna to Treat Rare Autoimmune Disease of the CNS

The U.S. Food and Drug Administration (FDA) has approved inebilizumab-cdon (Uplizna) to treat neuromyelitis optica spectrum disorder (NMOSD) in adult patients with the anti-AQP4 antibody. It is only the second therapy approved to treat the rare autoimmune disease of the central nervous system.

Approval was based on results of a clinical study of 230 adult patients, 213 of whom had antibodies against AQP4 (anti-AQPR antibody positive), that evaluated the efficacy and safety of intravenous Uplizna. During the 197-day study, the risk of an NMOSD relapse in the 161 anti-AQPR antibody positive patients who were treated with Uplizna was reduced by 77 percent when compared to the placebo treatment arm. There was no evidence of a benefit in patients who were anti-AQP4 antibody negative.

“Until recently, patients with NMOSD had no FDA-approved treatment options,” said Billy Dunn, MD, director of the office of neuroscience in the FDA’s Center for Drug Evaluation and Research. “Uplizna now represents the second approved therapy for these patients within the past year. We continue to remain highly committed to the development of additional safe and effective drugs for this rare and devastating disease.”