Ultomiris has been approved in the European Union as the first and only long-acting C5 complement inhibitor for the treatment of adult patients with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD).
“Just four years since delivering the first approved NMOSD treatment to the EU, we are pleased to once again advance the treatment landscape with Ultomiris, which may eliminate relapses for people impacted by NMOSD. This approval is the culmination of extensive work and collaboration with the NMOSD community, including patients, caregivers and healthcare providers who participated in the CHAMPION-NMOSD trial, and we’re proud to continue improving access to our innovative medicines worldwide,” says Marc Dunoyer, Chief Executive Officer, Alexion.
Based on results from the CHAMPION-NMOSD Phase III trial
The approval by the European Commission follows the positive opinion of the Committee for Medicinal Products for Human Use and is based on results from the CHAMPION-NMOSD Phase III trial, which were published online in the Annals of Neurology and selected as an abstract of distinction at the 2023 American Academy of Neurology Annual Meeting.2 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 and continuing through a median duration of 90 weeks.
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.
Regulatory submissions for Ultomiris for the treatment of NMOSD are also currently under review with multiple health authorities, including in the United States (US) and Japan.
Photo of Marc Dunoyer: Alexion