Ultomiris (ravulizumab) has been approved in the European Union (EU) 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).
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. 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.
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 5,000.
Orhan Aktas, MD, professor at the Department of Neurology, Medical Faculty at Heinrich-Heine-University, Düsseldorf, Germany, said: “Effective NMOSD management hinges on reducing relapses – even a single relapse may cause irreversible disabilities leaving patients unable to move, speak or care for themselves.
“This approval in Europe will offer patients and physicians a new, long-acting treatment with unmatched relapse risk reduction, as Ultomiris showed zero relapses in its pivotal trial with dosing every eight weeks.”
Marc Dunoyer, Chief Executive Officer, Alexion, said: “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.”
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.