Alexion has submitted a marketing application to the Food and Drug Administration (FDA) to extend the indication for Soliris (eculizumab) to be a treatment for patients with refractory generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive.

The application submission was supported by data form the Phase 3 REGAIN study. Soliris, a first-in-class terminal complement inhibitor, is currently approved to treat paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). It previously received Orphan Drug Designation for the treatment of patients with myasthenia gravis (MG). 

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MG is a debilitating neuromuscular disease in which patients suffer significant muscle weakness throughout the body. The segment of patients the new application is seeking to treat–refractory gMG patients who are anti-AChR antibody-positive – represents an ultra-rare population.

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