Regeneron Pharmaceuticals announced that cemdisiran, a small interfering RNA (siRNA) licensed from Alnylam, met pivotal endpoints in Phase III trials for generalized myasthenia gravis (MG). The siRNA therapy achieved substantial complement factor 5 reduction and improvement in symptom scores, positioning it for a planned FDA filing in early 2026. Cemdisiran monotherapy demonstrated a statistically meaningful clinical benefit compared to placebo, offering promise in this rare autoimmune neuromuscular disorder.<BR><BR>As the competition in the MG space intensifies, cemdisiran’s distinct mechanism and oral administration route may confer strategic advantages.