Regeneron Secures FDA Priority Review for Cemdisiran with Nov 2026 Decision
REGN•Regeneron’s cemdisiran NDA received FDA Priority Review with a November 2026 target action date and EMA MAA review underway for generalized myasthenia gravis. The filings, supported by Phase 3 NIMBLE data from a global trial of subcutaneous dosing every 12 weeks, also plan a Japan submission in early 2027.
1. Regulatory Submissions Accepted
The FDA has granted Priority Review to the New Drug Application for cemdisiran targeting adult patients with generalized myasthenia gravis, setting a November 2026 action date. The European Medicines Agency has also accepted the Marketing Authorization Application, with a decision expected in the second half of 2027.
2. Supporting Phase 3 NIMBLE Trial Data
Cemdisiran’s filing is supported by the Phase 3 NIMBLE trial, one of the largest global interventional studies in generalized myasthenia gravis, evaluating subcutaneous dosing every 12 weeks alongside standard immunosuppressants. Full efficacy and safety results were published in April 2026.
3. Next Steps and Market Outlook
Regeneron plans to submit regulatory filings in Japan in early 2027, aiming to launch cemdisiran as the first siRNA therapy offering quarterly dosing for gMG. With approximately 85,000 U.S. patients facing high unmet needs for durable, targeted treatments, approval could meaningfully expand Regeneron’s rare disease portfolio.
