Firdapse amifampridine phosphate regulatory update

Catalyst said FDA requested an additional “adequate and well-controlled” trial to support an NDA for Firdapse amifampridine phosphate to treat Lambert-Eaton myasthenic syndrome (LEMS). The request follows a meeting last month with FDA to discuss what the agency needs to accept the NDA, for which FDA issued a refusal to file letter in February (see BioCentury, Feb. 22). Catalyst said FDA is “open to discuss a study design that could efficiently accomplish the requirement with a small, short-term study.” The agency is also requesting “several more short-term toxicology studies, which are expected to start soon.” ...