The drug has already been approved for this indication in the EU, with regulatory reviews continuing elsewhere including in the US.
AstraZeneca has announced that Ultomiris (ravulizumab) has been approved in Japan as a 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 Japanese Ministry of Health, Labour and Welfare’s (MHLW) approval of the drug follows positive results from the CHAMPION-NMOSD phase 3 trial, recently published in the Annals of Neurology. In this trial, Ultomiris was compared to an external placebo group from the Soliris PREVENT clinical trial.
The drug met its primary endpoint of time to first on-trial relapse as confirmed by an independent adjudication committee. No relapses were reported in the group treated with Ultomiris with a median treatment duration of 73 weeks.
Ichiro Nakashima MD, professor at the Division of Neurology, Tohoku Medical and Pharmaceutical University, Japan, commented: “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, CEO of Alexion, added: “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.”
