Long-Term Data Support Ravulizumab for NMOSD

Final analysis of results from the long-term extension of the CHAMPION-NMOSD clinical trial (NCT04201262) showed that treatment with Ultomiris (ravulizumab; Alexion—AstraZeneca Rare Disease, Boston, MA) was associated with relapse prevention and stable or improved disability outcomes in adults with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD). These data were presented at the 41st Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS).

CHAMPION-NMOSD was a phase 3, open-label, externally placebo-controlled study. On day 1, participants received intravenous Ultomiris as a weight-based loading dose, followed by maintenance infusions on day 15 and every 8 weeks thereafter. After completing the primary treatment period, participants had the option to continue in the long-term extension for up to 2.5 years. Outcomes included time to first adjudicated on-trial relapse (OTR), risk reduction of relapse, disability scores, and safety. Fifty-five participants completed treatment in the long-term extension study.

Key results include the following:

• None of the participants treated with Ultomiris experienced an adjudicated OTR across 189.7 patient-years of follow-up.
• Compared with placebo, Ultomiris treatment was associated with a 98.9% relative reduction in risk of relapse (95% CI, 91.8 to 100; P< .0001).
• Hauser Ambulation Index scores remained stable in 81% of participants and improved in 13.8%.
• Expanded Disability Status Scale scores showed no clinically important worsening in 91.4%.
• Dose reductions or discontinuations occurred for 63% of participants who were receiving immunosuppressive therapy at baseline.
• The safety profile was consistent with previous studies, and most adverse events were mild and unrelated to treatment.