AstraZeneca's Ultomiris Nears Approval for NMOSD Treatment in EU
AstraZeneca's Ultomiris (ravulizumab) has received a recommendation for marketing authorization in the European Union (EU) for the treatment of adult patients who are anti-aquaporin-4 (AQP4) antibody positive (Ab+) and have neuromyelitis optica spectrum disorder (NMOSD).
If authorized, Ultomiris would become the first and only long-acting C5 complement inhibitor to be approved for the treatment of AQP4 Ab+ NMOSD in the EU.
Based on the results from the CHAMPION-NMOSD phase 3 trial, the positive recommendation for Ultomiris by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency was achieved. Ultomiris was compared to an external placebo arm from the pivotal Soliris PREVENT clinical trial in the CHAMPION-NMOSD trial.
The primary endpoint of time to first on-trial relapse was met by Ultomiris as confirmed by an independent adjudication committee. Notably, patients who received Ultomiris showed zero relapses with a median treatment duration of 73 weeks, indicating a 98.6% reduction in relapse risk (hazard ratio (95% CI): 0.014 (0.000, 0.103), P<0.0001). This benefit was sustained through a median duration of 90 weeks.
“Even one NMOSD relapse can lead to devastating long-term effects like vision loss, chronic pain and paralysis, which underscores the need for treatment innovations that help prevent relapses and optimize disease management," Orhan Aktas, MD, Professor at the Department of Neurology, Medical Faculty at Heinrich-Heine-University, Düsseldorf, Germany, said in the news release. "The sustained relapse risk reduction observed in the CHAMPION-NMOSD phase 3 trial supports the critical role this long-acting C5 complement inhibitor may have for the NMOSD community.”
The safety and tolerability of Ultomiris remained consistent with both previous clinical studies and real-world application. No fresh safety concerns were observed. COVID-19, headache, back pain, arthralgia, and urinary tract infection were the most frequently reported adverse events (AEs).
Every instance of COVID-19 was deemed non-serious and not linked to Ultomiris1. Ultomiris' regulatory submissions for NMOSD treatment are presently undergoing review by several health authorities, including those in the United States (US) and Japan.
Reference
1. Pittock SJ, Barnett M 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. American Academy of Neurology Annual Meeting, Abstract S5.002.
