AstraZeneca’s (AZ; NASDAQ: AZN) rare disease unit, Alexion, has registered its biologic Ultomiris (ravulizumab) with the US Food and Drug Administration (FDA) for the treatment of anti-AQP4 antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD) in adults. This registration follows an initial rejection by the FDA last year and positions Ultomiris as the only long-acting C5 complement inhibitor for this indication.
The decision was supported by data from a late-stage study that successfully met its primary endpoint, which was the time to relapse. The study recorded no relapse events in the experimental arm after a median treatment duration of 73 weeks, leading to a significant 98.6% reduction in relapse risk compared to a placebo control group. The study also reported no new safety signals associated with the use of Ultomiris.
As highlighted in the press release, Ultomiris offers a convenient 8-week dosing schedule, which is a significant advantage for patients. Prior to this approval, Ultomiris had already been approved in the US for the treatment of generalised myasthenia gravis (gMG), atypical haemolytic uraemic syndrome (aHUS), and paroxysmal nocturnal haemoglobinuria (PNH).- Flcube.com
