The FDA will review the New Drug Application (NDA) under Priority Review with a target action date in November 2026, following use of a Priority Review Voucher. A decision from the European Commission is anticipated in the second half of 2027.

The submissions are supported by data from the Phase 3 NIMBLE trial evaluating cemdisiran, dosed subcutaneously every 12 weeks, in adults with symptomatic gMG who may be receiving standard of care immunosuppressants based on the investigator’s discretion. Full data from NIMBLE, which is one of the largest global, interventional gMG trials conducted to date, were simultaneously published in The Lancet and presented at the American Academy of Neurology (AAN) Annual Meeting in April 2026. A regulatory filing in Japan is also planned for early 2027.

MG is a rare and chronic autoimmune disease where abnormal anti-AChR antibodies activate the complement system including C5, disrupting communication between nerves and muscles that results in debilitating and potentially life-threatening muscle weakness. Worldwide, an estimated 150 to 200 out of every million people have MG. In the U.S., the disease impacts approximately 85,000 people. Initial manifestations are usually ocular, but approximately 85% of MG patients experience progression to additional disease manifestations, which is then categorized as generalized MG. For these patients, the disease affects muscles throughout the body, resulting in extreme fatigue and difficulties with facial expression, speech, swallowing and mobility. For patients living with gMG, many continue to experience challenges with disease management including treatments that only address symptoms, long-term burden of immunosuppressants, lack of responsiveness as well as waning effectiveness, which can all affect their quality of life.

The safety and efficacy of cemdisiran, as well as its potential use for the treatment of gMG, are investigational and have not been fully evaluated or approved by any regulatory authority.

Regeneron is solely responsible for the development, manufacturing, and commercialization of cemdisiran as a monotherapy and in combination with C5 antibodies through a worldwide licensing agreement with Alnylam.