Regeneron Pharmaceuticals, licensing cemdisiran from Alnylam, announced successful pivotal trial results of the siRNA drug in generalized myasthenia gravis. The monotherapy reduced complement factor 5 levels and improved daily living activity scores in patients. Regeneron plans to file for FDA approval in early 2026. These results mark a promising advance in neuromuscular autoimmune disease treatment, with cemdisiran showing potential advantages over existing therapeutic classes.