Ravulizumab’s Long-Term Efficacy and Safety as NMOSD Treatment: Sean Pittock, MD


The director for the Mayo Clinic Center for Multiple Sclerosis and Autoimmune Neurology provided an overview of the open-label extension of the phase 3 CHAMPION-NMOSD trial and how the newly approved ravulizumab fits with other NMOSD treatments. [WATCH TIME: 4 minutes]

WATCH TIME: 4 minutes

"The benefit of ravulizumab over eculizumab is that ravulizumab is an infusion you get every 8 weeks, whereas eculizumab is an infusion you get every 2 weeks. So it’s a big difference for patients, they essentially only have to go into an infusion center about 6 times per year."

For years, there were no approved medications specific to patients with neuromyelitis optica spectrum disorder (NMOSD), a rare autoimmune disease of the central nervous system that primary affects that optic nerves and spinal cord. The field saw its first approval in 2019 with eculizumab (Soliris; Alexion), a compliment inhibitor therapy, for those with aquaporin-4 antibody-positive disease. Years later, ravulizumab (Ultomiris; Alexion), a terminal complement C5 inhibitor as well, became the fourth approved agent to treat patients with the condition, giving clinicians and patients a well-rounded toolbox of options.

At the 2024 American Academy of Neurology (AAN) Annual Meeting, held April 13-18, in Denver, Colorado, new interim data from the open-label extension of the CHAMPION-NMOSD trial (NCT04201262), the study that led to ravulizumab’s approval, was presented. In the phase 3 study, adults with NMOSD received intravenous, weight-based loading dose of ravulizumab on day 1 and a maintenance dose on day 15 and every 8 weeks thereafter. As of June 16, 2023, the median follow-up was 138.4 (range, 11.0-183.1) weeks for ravulizumab (n = 58), with 153.9 patient-years.

Led by Sean Pittock, MD, the open-label data was consistent with outcomes seen in the primary treatment period, as no patients on ravulizumab experienced an adjundicated relapse and most demonstrated stable or improved disability measures. Pittock, director for the Mayo Clinic Center for Multiple Sclerosis and Autoimmune Neurology, sat down with NeurologyLive® at the meeting to discuss the findings and how they add to the efficacy and safety profile of ravulizumab. Furthermore, he spoke on some of the differences and advantages of this therapy over other previously approved medications for NMOSD.

Click here for more AAN 2024 coverage.

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 (AQP4+ NMOSD): interim analysis from the ongoing phase 3 CHAMPION-NMOSD trial. Presented at: 2024 AAN Annual Meeting; April 13-18; ABSTRACT 002489
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