Alexion Pharmaceuticals sBLA Submitted on Soliris

FDA has accepted for review an Alexion Pharmaceuticals supplemental BLA seeking to expand the indication for Soliris  (eculizumab) as a treatment for patients with refractory generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor antibody-positive. Soliris is described as a first-in-class terminal complement inhibitor, and is approved in the U.S. for treating patients with paroxysmal nocturnal hemoglobinuria to reduce hemolysis. The submission is based on data from the Phase 3 REGAIN study. The agency has set a user fee review target date of 10/23.

Refractory gMG is an ultra-rare disease, according to the company. “Despite existing treatment options for gMG, patients with refractory gMG continue to face severe complications, including difficulty walking, talking, swallowing, and breathing normally,” it says. “Exacerbations of their disease may be life-threatening and require hospitalization and intensive care.”