Regeneron’s RNA Med for Muscle Disease Meets Trial Goals; FDA Filing Planned for Next Year

A Regeneron Pharmaceuticals drug that lowers levels of a disease-driving protein has met the goals of a pivotal test in the rare disease generalized myasthenia gravis. Based on these results, Regeneron plans to seek FDA approval for the therapy, potentially introducing a new mechanism of action to the increasingly competitive field of treatments for this neuromuscular disorder.

The Regeneron drug, cemdisiran, takes a new approach to the treatment of myasthenia gravis, a disease that leads to difficulty swallowing as well as muscle weakness. The autoimmune disorder develops as the body attacks proteins key to communication between nerves and muscles. Abnormal antibodies produced by the body activate the complement system, a part of the immune system. Cemdisiran is a small interfering RNA