AstraZeneca Ultomiris gets approval in Japan to treat rare neuromuscular disorder
Marcelo Ricardo Daros


The Japanese Ministry of Health, Labour and Welfare approved AstraZeneca's (NASDAQ:AZN) Ultomiris to treat adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive and whose symptoms are difficult to control with high-dose intravenous immunoglobulin therapy (IVIg) or plasmaphaeresis.

Japanâs Pharmaceuticals and Medical Devices Agency also indicated that Ultomiris can also be used for patients who cannot receive high-dose IVIg or plasmaphaeresis due to complications, adverse reactions or other limiting factors, the company said in an Aug. 25 press release.
The British pharma giant said Ultomiris is the first and only long-acting C5 complement inhibitor to be approved in Japan.
The approval was backed by data from a phase 3 trial called CHAMPION-MG.
Ultomiris was approved in the U.S. for adults with anti-AChR antibody-positive gMG in April.
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.