Positive Results for Soliris in Neuromyelitis Optica Spectrum Disorder Trial
Treatment with Soliris (eculizumab; Alexion) was associated with reduced risk of relapse in patients with anti-aquaporin-4 (AQP4) auto antibody-positive neuromyelitis optica spectrum disorder (NMOSD), according to topline results from the Phase 3 PREVENT study. NMOSD is a rare, complement-mediated central nervous system disorder characterized by relapses that result in severe disability (blindness, paralysis) and potentially premature death.
In the PREVENT study, the safety and efficacy of Soliris was compared to placebo in patients with AQP4 auto antibody-positive NMOSD (N=143). The primary endpoint of the trial was time to first on-trial relapse as adjudicated by an independent committee; a key secondary endpoint was adjudicated on-trial annualized relapse rate.
Results showed that Soliris reduced the risk of NMOSD relapse by 94.2% compared with placebo (P <.0001); 97.9% of Soliris-treated patients were relapse-free at 48 weeks compared with 63.2% of patients in the placebo arm. Moreover, compared with placebo, treatment with Soliris reduced the adjudicated on-trial annualized relapse rate by 95.5% (P <.0001). Soliris was generally well-tolerated with a safety profile similar to that seen in previous studies and real-world use.
“The primary goal in treating NMOSD is relapse prevention as each relapse further increases disability, which makes this disease so devastating,” said Michael Levy, MD, PhD, Associate Professor at Johns Hopkins University, and Director of the Neuromyelitis Optica Clinic in Baltimore, MD. “The substantial effect of Soliris seen in this groundbreaking randomized, controlled study in NMOSD could potentially become a turning point for patients and their families who live in constant fear of relapse.”
Soliris, a complement inhibitor, is currently approved by the Food and Drug Administration (FDA) for the treatment of paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome, and for adult patients with generalized myasthenia gravis who are anti-acetylcholine receptor (AchR) antibody positive.
“These results far exceeded our expectations, said John Orloff, MD, Executive Vice President and Head of Research & Development at Alexion “Given that patients currently have no approved therapies [for NMOSD], we are moving quickly to discuss these results with regulators and file for approval in the US, EU, and Japan.”
For more information visit Alexion.com.