The Food and Drug Administration (FDA) has approved Soliris (eculizumab; Alexion Pharmaceuticals, Inc) for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive.

The approval was based on the phase 3, 48-week, double blind, placebo controlled PREVENT study that evaluated the efficacy of Soliris for the treatment of NMOSD in 143 patients aged 19 to 75 years who were anti-AQP4 antibody positive. Patients were randomized to receive either Soliris (N=96) or placebo (N=47). Additionally, 76% of patients received concomitant immunosuppressive therapy (IST), including chronic corticosteroids, while 24% of patients did not receive concomitant treatment.

Results showed the time to the first adjudicated on-trial relapse (primary end point) was statistically significantly longer in the Soliris-treated patients compared with the placebo arm (relative risk reduction 94%; hazard ratio [HR] 0.058; P <.0001); concomitant IST did not affect the primary end point. At 48 weeks, 97.9% of Soliris-treated patients were relapse free compared with 63.2% of patients receiving placebo. 

Regarding safety, the most common treatment-emergent adverse events were upper RTI, nasopharyngitis, diarrhea, back pain, dizziness, influenza, arthralgia, pharyngitis, and contusion. Soliris has a boxed warning for severe meningococcal infections and is only available under a Risk Evaluation and Mitigation Strategy (REMS) program that prescribers are required to enroll in.

“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.”

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Soliris, a complement inhibitor, is already indicated for treatment of paroxysmal nocturnal hemoglobinuria to reduce hemolysis, atypical hemolytic uremic syndrome to inhibit complement-mediated thrombotic microangiopathy, and generalized myasthenia gravis in adults who are anti-acetylcholine receptor antibody positive.

The product is supplied in 30mL single-dose vials containing 10mg/mL strength preservative-free solution for intravenous (IV) infusion after dilution.

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