Catalyst Will Try To Salvage Rare Disease Drug from Failed Phase III Trial
Executive Summary
Despite a Phase III study that found no statistically significant outcomes for its endpoints for Firdapse, Catalyst will seek FDA approval for treating a subgroup of patients who showed improvement
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Investors hit Catalyst Pharmaceuticals Inc. hard on Feb. 17, driving shares down 50%, after the company disclosed the FDA refused to file (RTF) the Florida firm's new drug application (NDA) for Firdapse (amifampridine phosphate), a 3,4-diaminopyridine phosphate, as a symptomatic treatment for two very rare neuromuscular diseases – Lambert Eaton myasthenic syndrome (LEMS) and congenital myasthenic syndromes (CMS).