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: “As a single NMOSD relapse can result in long-term and life-altering disability, relapse prevention is the primary treatment goal in this disease and essential to help patients maintain quality of life.
“With no relapses observed in the pivotal CHAMPION-NMOSD trial, the approval of this long-acting C5 complement inhibitor in Japan is a significant advance for patients with AQP4 Ab+ NMOSD, offering dosing every eight weeks and the potential to live relapse-free.”
Marc Dunoyer, Chief Executive Officer, Alexion, said: “Alexion transformed the NMOSD landscape by uncovering the exceptional efficacy of C5 complement inhibition in reducing the risk of relapses for patients.
“With today’s approval, we continue to deliver on our commitment to the NMOSD community, offering patients an innovative long-acting treatment option that has the potential to eliminate relapses with convenient dosing every eight weeks. We are proud to expand the reach of Ultomiris as we work to improve patients’ lives around the world.”
Overall, the safety and tolerability of Ultomiris in the CHAMPION-NMOSD trial were consistent with previous clinical studies and real-world use, and no new safety signals were observed. The most common adverse events (AEs) were COVID-19, headache, back pain, arthralgia and urinary tract infection. All cases of COVID-19 were non-serious and considered to be unrelated to Ultomiris.
Ultomiris was recently approved in the European Union (EU) for the treatment of adult patients with NMOSD who are AQP4 Ab+, and regulatory reviews are ongoing in additional countries, including in the United States (US).