C5 Combo (pozelimab and cemdisiran) - (NIMBLE)

June 22, 2026: Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) announced that the FDA and EMA have accepted regulatory applications for cemdisiran for treatment of adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive. The FDA accepted the New Drug Application (NDA) under Priority Review (using a Priority Review Voucher) with a target action date in November 2026. The EMA has accepted the submission; a European Commission decision is anticipated in the second half of 2027. Regeneron also plans a regulatory filing in Japan in early 2027.

The filings are supported by the Phase 3 NIMBLE trial of subcutaneous cemdisiran dosed every 12 weeks in adults with symptomatic gMG (patients could receive standard-of-care immunosuppressants at investigator discretion). Full NIMBLE data were published in The Lancet and presented at the American Academy of Neurology (AAN) Annual Meeting in April 2026. Cemdisiran remains investigational and not yet approved. Regeneron is responsible for development, manufacturing, and commercialization under a worldwide licensing agreement with Alnylam.