Regulatory Agencies Accept Cemdisiran Applications for Myasthenia Gravis Treatment

Regeneron Pharmaceuticals has announced that both the FDA and EMA have accepted regulatory submissions for cemdisiran, an investigational treatment for generalized myasthenia gravis. The FDA granted Priority Review, with a decision expected by November 2026. This therapy could be the first siRNA approved for gMG and potentially offer convenient quarterly subcutaneous dosing.