Ultomiris approved in the US for the treatment of adults with neuromyelitis optica spectrum disorder (NMOSD).- AstraZeneca

The approval by the FDA was based on positive results from the CHAMPION-NMOSD Phase III trial, which were published in the Annals of Neurology (previously cited). 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).></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 US is estimated at approximately 6,000.