Ultomiris (ravulizumab) has been approved in Japan as the first and only long-acting C5 complement inhibitor for the prevention of relapses in patients with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD), including neuromyelitis optica.
The approval by the Japanese Ministry of Health, Labour and Welfare (MHLW) was based on positive results from the CHAMPION-NMOSD phase III trial, which were recently published in the Annals of Neurology and selected as an abstract of distinction at the 2023 American Academy of Neurology Annual Meeting. In the 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. 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 Japan is estimated at approximately 4,000.
Ichiro Nakashima, MD, Professor at the Division of Neurology, Tohoku Medical and Pharmaceutical University, Sendai, Japan said:
