AstraZeneca Ultomiris gets approval in EU to treat rare neuromuscular disorder
artJazz

The European Commission (EC) approved the expanded use of AstraZeneca's (NASDAQ:AZN) Ultomiris (ravulizumab) as an add-on to standard therapy for adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive.
A panel of the European Medicines Agency (EMA) had recommended the approval of the drug in July.
The decision marked the first and only approval for a long-acting C5 complement inhibitor to treat of gMG in Europe, the company said in a Sept. 23 press release.
GMG is a rare neuromuscular disorder characterized by loss of muscle function and weakness, causing slurred speech, difficulty in eating and moving, among other things.
The EC approval was backed by data from a phase 3 trial called CHAMPION-MG.
The British pharma giant noted that Ultomiris â which was inherited by AstraZeneca via the acquisition of Alexion â was approved in the U.S. in April, and in Japan in August for certain adults with gMG.
Ultomiris is also approved in the U.S., EU and Japan to treat certain adults with a type of blood disorder called paroxysmal nocturnal haemoglobinuria (PNH) and for certain children with PNH in the U.S. and EU, the company added.