Success of Ravulizumab Through the Phase 3 CHAMPION-NMOSD Trial: Sean Pittock, MD

WATCH TIME: 5 minutes

"The results were staggering. The good news for patients is that we had zero clinical relapses in the 74 weeks of follow-up in the open-label phase of the trial. That resulted in a 98.6% risk reduction, which is great news for patients."

CHAMPION-NMOSD (NCT04201262) was a phase 3, open-label, externally controlled interventional study that assessed the efficacy and safety of the terminal complement inhibitor ravulizumab (Ultomiris; AstraZeneca) in adults with aquaporin-4-antibody-positve neuromyelitis optica spectrum disorder (NMSOD). Ravulizumab binds the same complement component 5 epitope as eculizumab (Soliris; Alexion), a previously approved therapy for NMOSD, but has a longer half-life, enabling an extended dosing interval of 8 weeks instead of 2.

The trial comprised of individuals who had a history of at least 1 relapse in the 12 months before screening, and an Expanded Disability Status Scale score of less than 7. Following a median follow-up of 73.5 weeks on treatment, the primary end point was met, with no patients demonstrating adjudicated relapses on ravulizumab vs 20 with placebo (relapse risk reduction, 98.6%; P <.0001). Presented at the 2023 American Academy of Neurology (AAN) Annual Meeting, held April 22-27, in Boston, Massachusetts, fewer patients experienced clinically important Hauser Ambulation Index score worsening with ravulizumab (3.4%) than placebo (23.4%; P = .023).

At the meeting, lead investigator Sean Pittock, MD, sat down with NeurologyLive^® to discuss the overall design of the trial, and why ravulizumab, a previously approved therapy for paroxysmal nocturnal hemoglubinuria, is beneficial for patients with NMOSD. Pittock, director of the Mayo Clinic Center for Multiple Sclerosis and Autoimmune Neurology, detailed some of the major take-home points clinicians in NMOSD and other related fields should be aware of.

REFERENCE
1. Pittock S, Barnett M, Bennett J, et al. Efficacy and safety of ravulizumab in adults with anti-aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder: outcomes from the phase 3 CHAMPION-NMOSD trial. Presented at: 2023 AAN Annual Meeting; April 22-27; Boston, MA. Abstract 002965