Positive high-level results were announced from a phase 3 trial evaluating ravulizumab-cwvz in adults with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD). 

The open-label, multicenter, external placebo-controlled CHAMPION-NMOSD trial (ClinicalTrials.gov Identifier: NCT04201262) evaluated the efficacy and safety of ravulizumab-cwvz, a C5 complement inhibitor, in 58 adults with AQP4 Ab+ NMOSD. All patients received a single weight-based loading dose of ravulizumab-cwvz on day 1, followed by regular weight-based maintenance dosing every 8 weeks starting on day 15. 

Due to the potential long-term functional impact of NMOSD relapses, ravulizumab-cwvz was compared with the external placebo arm from the phase 3 Soliris® PREVENT trial (ClinicalTrials.gov Identifier: NCT01892345). The primary endpoint was time to first adjudicated on-trial relapse, as confirmed by an independent adjudication committee.

Results demonstrated that treatment with ravulizumab-cwvz met the primary endpoint demonstrating a statistically significant and clinically meaningful reduction in the risk of relapse compared with the external placebo arm. Over a median treatment duration of 73 weeks, there were no relapses observed among patients treated with ravulizumab-cwvz.

The safety profile of ravulizumab-cwvz was consistent with that seen in previous studies and other approved indications. Fifty-six patients are continuing treatment with ravulizumab-cwvz in an ongoing long-term extension period of the study.

Additional study data will be presented at a forthcoming medical meeting and submitted to global health authorities.

Ravulizumab-cwvz is currently marketed under the brand name Ultomiris and is approved for the treatment of paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome.

“Soliris established the role of complement inhibition in preventing relapses in NMOSD, and with Ultomiris, we continue to innovate for patients with a more convenient every 8-week dosing schedule,” said Marc Dunoyer, Chief Executive Officer, Alexion. “These trial results show that Ultomiris may help patients move towards eliminating relapses, which is an important advancement in the treatment of NMOSD.”


Ultomiris® (ravulizumab-cwvz) met primary endpoint in CHAMPION-NMOSD phase iii trial in adults with neuromyelitis optica spectrum disorder. News release. AstraZeneca. Accessed May 5, 2022. https://www.businesswire.com/news/home/20220505005260/en/ULTOMIRIS%C2%AE-ravulizumab-cwvz-Met-Primary-Endpoint-in-CHAMPION-NMOSD-Phase-III-Trial-in-Adults-with-Neuromyelitis-Optica-Spectrum-Disorder