AstraZeneca's Ultomiris wins FDA approval for rare neuromuscular disorder
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The U.S. Food and Drug Administration approved AstraZeneca's (NASDAQ:AZN) Ultomiris to treat adult patients with generalised myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive.
GMG is a rare neuromuscular disorder characterized by loss of muscle function and weakness, causing slurred speech, difficulty in eating and requiring assistance in moving, among other things.

AstraZeneca's (AZN), which inherited Ultomiris via the acquisition of Alexion, said the approval was backed by data from a phase 3 trial, dubbed CHAMPION-MG.
The British drugmaker noted that the FDA action for Ultomiris (ravulizumab-cwvz) marks the first and only approval for a long-acting C5 complement inhibitor for treating gMG.
AstraZeneca (AZN) noted that regulatory submissions for Ultomiris to treat gMG are currently under review with multiple health authorities, including in the EU and Japan.