FDA Approves First Drug for Neuromyelitis Optica

Deborah Brauser – DISCLOSURES 

June 27, 2019

The US Food and Drug Administration (FDA) has approved the humanized monoclonal antibody eculizumab (Soliris, Alexion Pharmaceuticals) for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adults who test positive for anti-aquaporin-4 (AQP4) autoantibodies, the agency announced in a statement earlier today.

This makes eculizumab the first FDA-approved drug for this indication.

“This approval changes the landscape of therapy for patients with NMOSD,” Billy Dunn, MD, director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research, said in a news release.

He added that NMOSD is a “debilitating disease that profoundly impacts patients’ lives.”

The approval was based in large part on results from the phase 3 multicountry PREVENT trial, which were presented at the recent American Academy of Neurology (AAN) 2019 Annual Meeting and were simultaneously published online in the New England Journal of Medicine.

As reported at the time by Medscape Medical News, 43% of the 143 participants with AQP4 antibody-positive NMOSD who received placebo experienced adjudicated relapses, vs 3% of those who received intravenous eculizumab. For the latter group, annualized relapse rates were also lower.

Asked to comment on the approval, Eddie Patton, MD, neurologist and clinical assistant professor in the Department of Neurology and Neurosurgery, University of Texas, Houston, noted that the drug “gives us a very focused treatment” for patients with NMOSD.

“I can’t underscore enough the burden that this disease typically has on patients. So it’s exciting to have something that’s targeted and has been shown to be more effective, as far as reducing relapses,” Patton told Medscape Medical News.

Eculizumab is a C5 complement inhibitor.

NMOSD, which is also often referred to as NMO, is a rare, complement-mediated disorder of the central nervous system. It is characterized by relapses that are associated with increasing disability, including blindness and paralysis. The FDA notes that about 4000 to 8000 patients are affected by NMOSD in the United States.

In addition, about 73% of patients with the disorder have anti-AQP4 antibodies. NMOSD primarily affects women.