The U.S. Food and Drug Administration (FDA) has approved Ultomiris (ravulizumab-cwvz) for the treatment of adult patients with Neuromyelitis Optica Spectrum Disorder (NMOSD) that tests positive for anti-aquaporin-4 (AQP4 Ab+). This approval is a major milestone as it makes Ultomiris the first and only long-acting C5 complement inhibitor available for NMOSD treatment.
Neuromyelitis optica spectrum disorder (NMOSD) affects the spinal cord and optic nerves, occurring when the immune system mistakenly attacks healthy cells. The cause of NMOSD is unknown. Symptoms include pain, weakness, bowel and bladder problems, and temporary vision loss. Some people may develop permanent muscle weakness and vision loss. NMOSD usually occurs in adulthood, but symptoms may start at any age. Symptoms may appear as a single attack lasting months, or come and go over time.
Ultomiris (ravulizumab-cwvz) is the first and only long-lasting C5 complement inhibitor, targeting C5 protein in the terminal complement cascade. This reduces the level of which the body attacks healthy cells. Ravulizumab-cwvz is administered every eight weeks in an intravenous injection.
The approval comes from the positive outcomes from the CHAMPION-NMOSD Phase III trial, an open-label, multicenter trial evaluating the safety and efficacy of ravulizumab-cwvz in adults with NMOSD. The trial demonstrated that ravulizumab-cwvz met the primary endpoint of relapse prevention. Impressively, no relapses were observed among ravulizumab-cwvz 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). Safety and tolerability were also observed.
For more information on NMOSD and other rare ophthalmology disorders, visit https://checkrare.com/diseases/ophthalmology-eye-diseases/
