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

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

Cemdisiran, a small interfering RNA drug that Regeneron Pharmaceuticals licensed from Alnylam Pharmaceuticals, met the goals of its pivotal test in generalized myasthenia gravis. The rare neuromuscular disease has several new therapies, but analysts see cemdisiran as having advantages over one particular class of drugs. The post Regeneron’s RNA Med for Muscle Disease Meets Trial Goals; FDA Filing Planned for Next Year appeared first on MedCity N…

RNA drug from Regeneron, Alnylam succeeds in rare autoimmune disease trial

Cemdisiran helped control symptoms of generalized myasthenia gravis, but combination treatment with a Regeneron antibody didn't appear as effective.

Regeneron tees up myasthenia gravis drug approval after Phase III victory

If approved, Regeneron's siRNA therapy will join the myasthenia gravis market alongside AstraZeneca’s dominant drugs Ultomiris and Soliris.The post Regeneron tees up myasthenia gravis drug approval after Phase III victory appeared first on Clinical Trials Arena.

Regeneron’s siRNA Therapy to Myasthenia Gravis Delivers in Phase III Trial

When designing an inhibitory drug, is the absolute maximum achievable inactivation of a therapeutic target always necessary? For Regeneron Pharmaceuticals, the Phase III NIMBLE trial of its siRNA therapy targeting complement component 5 (C5), cemdisiran, in generalized myasthenia gravis (gMG) suggests the answer may be no. In fact, in some cases, incomplete inhibition may even be better. In patients with gMG—an autoimmune disorder that affects t…

Regeneron claims Phase 3 win for a lesser-known rare disease drug

Regeneron on Tuesday declared a Phase 3 success for an under-the-radar drug it licensed from Alnylam, and it plans to take the program to the FDA early next year. In a study of about 200 ...