Catalyst Will Try To Salvage Rare Disease Drug from Failed Phase III Trial
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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Lawsuit seeking invalidation of rare disease treatment's approval in Lambert-Eaton myasthenic syndrome encompasses policy trifecta of exclusivity, drug pricing, and expanded access issues.
Catalyst Collapses On FDA Firdapse Refusal
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).