The European Commission Approves Ultomiris for Treatment of Adults with AQP4+ NMOSD in EU
The European Commission has approved the first C5 complement inhibitor for the treatment of adults with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD) in the European Union (EU). Approval is based on results from the CHAMPION-NMOSD phase 3 trial (NCT04201262) which demonstrated zero relapses among patients treated with Ultomiris (ravulizumab-cwvz; AstraZeneca, Boston, MA) with a median treatment duration ranging from 73 to 93 weeks (relapse risk reduction: 98.6%, hazard ratio [95% CI]: 0.014 [0.000, 0.103], P<.0001) compared with those taking a placebo.
The CHAMPION-NMOSD trial included 58 participants from across North America, Europe, Asia-Pacific, and Japan who met the following requirements: a confirmed NMOSD diagnosis, a positive anti-AQP4 antibody test, at least 1 attack or relapse in the 12 months prior to the screening visit, an Expanded Disability Status Scale score of 7 or less, and body weight of at least 40 kg at trial entry. All participants received a weight-based dose of Ultomiris beginning on day 1 followed by an additional weight-based dosage every 8 weeks beginning on day 15. Efficacy and safety outcomes of those treated with Ultimoris were compared with results obtained from the external placebo arm of the PREVENT clinical trial (NCT01892345).
Approval of Ultimoris in the EU marks an important step for treatment of this rare condition, as relapse of neurologic symptoms is a crucial barrier to the NMOSD recovery process. As Orhan Aktas, MD, Professor at the Department of Neurology, Medical Faculty at Heinrich-Heine-University, Düsseldorf, Germany, notes: “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.”