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FDA Expands AstraZeneca's Indication for C5 Protein Inhibitor Ultomiris

NEW YORK – The US Food and Drug Administration has expanded the indication of Ultomiris (ravulizumab-cwvz) to adult patients with anti-aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder (NMOSD), a rare autoimmune disease, AstraZeneca said Monday.

The drug, a C5 complement inhibitor, is already approved in the US for treating anti-acetylcholine receptor antibody-positive generalized myasthenia gravis for paroxysmal nocturnal hemoglobinuria or atypical hemolytic uremic syndrome. The new indication approves it as a treatment for the three-quarters of patients with NMOSD who specifically produce antibodies that bind to the aquaporin-4 protein, which activates the complement system and hinders the body's ability to fight infection.

The drug is dosed every eight weeks and is designed to eliminate NMOSD relapses.

"C5 inhibition has been proven to offer efficacy in reducing the risk of NMOSD relapses by blocking the complement system, a part of the immune system, from attacking healthy cells in the spinal cord, optic nerve, and brain," Sean Pittock, director of the center for multiple sclerosis and autoimmune neurology and the neuroimmunology laboratory at the Mayo Clinic, said in a statement. Pittock was also the lead primary investigator in the pivotal Phase III CHAMPION-NMOSD trial of Ultomiris, data from which supported this latest FDA approval.

In that trial, researchers compared patients treated with Ultomiris against an external placebo arm from the Phase III PREVENT trial of Soliris (eculizumab), another NMOSD treatment sold by AstraZeneca. Ultomiris met the primary endpoint of time to first on-trial relapse, with no relapses observed among Ultomiris-treated patients for a median treatment duration of 73 weeks.

The most common adverse events seen with Ultomiris treatment were COVID-19, headache, back pain, arthralgia, and urinary tract infection, which was consistent with previous clinical studies and real-world use.

Ultomiris is already approved for certain patients with NMOSD in Japan and Europe.