Posted by Michael Wonder on 29 Nov 2018
FDA approves first treatment for Lambert-Eaton myasthenic syndrome, a rare auto-immune disorder
28 November 2018 - The U.S. FDA today approved Firdapse (amifampridine) tablets for the treatment of Lambert-Eaton myasthenic syndrome in adults.
LEMS is a rare autoimmune disorder that affects the connection between nerves and muscles and causes weakness and other symptoms in affected patients. This is the first FDA approval of a treatment for LEMS.
The efficacy of Firdapse was studied in two clinical trials that together included 64 adult patients who received Firdapse or placebo. The studies measured the Quantitative Myasthenia Gravis score (a 13-item physician-rated categorical scale assessing muscle weakness) and the Subject Global Impression (a seven-point scale on which patients rated their overall impression of the effects of the study treatment on their physical well-being). For both measures, the patients receiving Firdapse experienced a greater benefit than those on placebo.
