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Catalyst Stumbles With Filing Of Controversial 'Breakthrough Therapy' Firdapse

 
• By Emily Hayes

FDA’s refuse-to-file letter for Firdapse in two ultra-rare neuromuscular disorders does not make judgments about the drug’s safety or efficacy, says Catalyst.

Catalyst Pharmaceutical Partners Inc.says it is working with FDA on a path for resubmitting its orphan disease drug Firdapse after receiving a “refuse-to-file” letter for an application submitted in December, and notes that there has been no judgment about the drug’s safety or efficacy.

The company announced on Feb. 17 it had received a refuse-to-file letter for the drug in two indications, both ultra-rare neuromuscular disorders: Lambert-Eaton myasthenic syndrome (LEMS) and Congenital myasthenic syndromes...

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