Regeneron's Cemdisiran Gets FDA Priority Review for gMG
Regeneron announced that the FDA and EMA have accepted regulatory applications for cemdisiran to treat anti-AChR antibody-positive gMG, with FDA granting Priority Review and a target action date in November 2026. If approved, it would be the first siRNA therapy for gMG, administered subcutaneously only four times a year.
Key Numbers
Regeneron Pharmaceuticals (REGN) announced that the U.S. FDA and European Medicines Agency (EMA) have accepted regulatory applications for cemdisiran to treat adult patients with anti-AChR antibody-positive generalized myasthenia gravis (gMG). The FDA granted Priority Review with a target action date in November 2026.
The Product
Cemdisiran is an investigational small interfering RNA (siRNA) therapy administered subcutaneously. If approved, it would be the first siRNA therapy for gMG and the only option given subcutaneously just four times a year, potentially reshaping treatment convenience in this rare autoimmune disease.
Pricing and Availability
Pricing details have not been disclosed by Regeneron. Final approval depends on the regulatory review outcome.
Competition
Current gMG treatments include cholinesterase inhibitors, corticosteroids, immunosuppressants, and monoclonal antibodies like eculizumab and ravulizumab. Cemdisiran offers a novel mechanism (siRNA) and less frequent dosing.
Potential Impact on the Company
If approved, cemdisiran could strengthen Regeneron's rare disease portfolio and provide a new revenue stream. It may also shift the narrative around the company from reliance on eye drugs to expansion into neuromuscular disorders.
Frequently Asked Questions
Found this useful? Share it