What's Happening?
Regeneron has announced positive Phase III results for its siRNA therapy cemdisiran in patients with generalized myasthenia gravis. The study demonstrated significant improvements in disease activity and daily living scores, paving the way for a regulatory filing in early 2026. Cemdisiran, developed in collaboration with Alnylam, targets complement factor 5 to suppress the complement cascade.
Why It's Important?
The success of cemdisiran in Phase III trials represents a potential advancement in the treatment of myasthenia gravis, offering a new therapeutic option for patients. Regeneron's planned filing could lead to the approval of a novel siRNA therapy, impacting the landscape of neuromuscular disease treatment.
