Drugs

Catalyst Wants Specific Tests for Some Amifampridine NDAs

David McFarland

— 1 min read
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Catalyst Pharmaceuticals is petitioning FDA to require blinded, controlled trials for approval of an NDA or sNDA for any amifampridine drug product intended to treat genetically confirmed cases of congenital myasthenic syndrome (CMS). The 6/14 petition specifically asks that FDA: 

  •          instruct review divisions in which a pending NDA for the use of amifampridine to treat CMS of genetic origin to not consider such application approvable based on a mechanistic argument of efficacy, and that the application include well-controlled randomized clinical trials as evidence of efficacy and safety to be considered approvable;
  •          issue guidance recommending seven technical items stated by Catalyst in the petition.

Catalyst says its NDA for Firdapse (amifampridine) 10 mg tablets for treating adults with Lambert-Eaton myasthenic syndrome was approved by FDA in 2018.

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