Regeneron's investigational drug cemdisiran, administered subcutaneously every 12 weeks, demonstrated rapid and sustained improvements in muscle function and daily activities in adults with generalized myasthenia gravis (gMG) in the Phase 3 NIMBLE trial. The trial met its primary and key secondary endpoints at 24 weeks, showing clinically meaningful benefits compared to placebo with a favorable safety profile. Cemdisiran could become the first siRNA therapy approved for gMG, with a U.S. regulatory application already submitted and further filings planned in 2026. This treatment offers a convenient dosing schedule and potential to transform gMG care by reducing treatment burden and improving patient quality of life.
