Ultomiris approved in EU to treat anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder .- AstraZeneca

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 (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.></0.0001)>

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.”

See- "Ravulizumab in Aquaporin-4–Positive Neuromyelitis Optica Spectrum Disorder": Sean J. Pittock MD, Michael Barnett MD, PhD, Jeffrey L. Bennett MD, PhD, Achim Berthele MD, Jérôme de Sèze PhD, Michael Levy MD, PhD, Ichiro Nakashima MD, PhD … First published: Annals of Neurology: 03 March 2023 https://doi.org/10.1002/ana.26626.