• 04 Mar 2023
• Admin
• News Article

EMA committee recommends marketing approval for Ultomiris to treat adults with neuromyelitis optica spectrum disorder

Ultomiris (ravulizumab) has been recommended for marketing authorisation in the European Union (EU) for the treatment of adult patients with neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 (AQP4) antibody positive (Ab+). If authorised, Ultomiris would be the first and only approved long-acting C5 complement inhibitor for the treatment of AQP4 Ab+ NMOSD in the EU. The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency based its positive opinion on results from the CHAMPION-NMOSD phase III trial. In the CHAMPION-NMOSD trial, Ultomiris was compared to an external placebo arm from the pivotal Soliris PREVENT clinical trial. Ultomiris met the primary endpoint of time to first on-trial relapse as confirmed by an independent adjudication committee. Notably, data showed zero relapses were observed among Ultomiris patients with a median treatment duration of 73 weeks (relapse risk reduction: 98.6%, hazard ratio (95% CI): 0.014 (0.000, 0.103), p<0.0001), and continuing through a median duration of 90 weeks. NMOSD is a rare and debilitating autoimmune disease that affects the central nervous system (CNS), including the spine and optic nerves. Most people living with NMOSD experience unpredictable relapses, characterised by a new onset of neurologic symptoms or worsening of existing neurologic symptoms, which tend to be severe and recurrent and may result in permanent disability. The diagnosed prevalence of adults with NMOSD in the EU is estimated at approximately 6,000. Orhan Aktas, MD, Professor at the Department of Neurology, Medical Faculty at Heinrich-Heine-University, D