US FDA appoves eculizumab for neuromyelitis opticaJune 28, 2019
The US Food and Drug Administration (FDA) has approved the humanized monoclonal antibody eculizumab (Soliris) for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adults.
The intravenous drug, eculizumab,was approved for patients who are positive for anti-aquaporin-4 (AQP4) autoantibodies, the agency announced. This is the first approved treatment for NMOSD..
“Soliris provides the first FDA-approved treatment for neuromyelitis optica spectrum disorder, a debilitating disease that profoundly impacts patients’ lives,” said Billy Dunn, M.D., director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research, in a news release.
NMOSD is an autoimmune disease of the central nervous system that mainly affects the optic nerves and spinal cord. This rare, complement-mediated disorder of the CNS is characterized by relapses that are associated with increasing disability, including blindness and paralysis.
“This approval changes the landscape of therapy for patients with NMOSD,”said Dunn.
The approval was largely based on results from the phase 3 multicountry PREVENT trial. Over 48 weeks, the treatment with eculizumab reduced the number of NMOSD relapses by 94% compared with placebo treatment, reported FDA.
The results were presented at the recent American Academy of Neurology (AAN) 2019 Annual Meeting and were published online in the New England Journal of Medicine.
Eculizumab will be available only through a risk evaluation and mitigation strategy programme and must be dispensed with the FDA-approved patient Medication Guide, the FDA said.