Catalyst Pharmaceuticals has received a "Refusal to File" letter from the U.S. FDA in response to the company's New Drug Application for Firdapse.
Firdapse is meant to treat Lambert-Eaton myasthenic syndrome (LEMS) and congenital myasthenic syndromes (CMS) -- rare, muscle-wasting disorders.
The FDA letter states that after a preliminary review, the agency has found that the application, which was submitted in December 2015, was not sufficiently complete, and requests additional supporting information. The letter does not provide comment on the acceptability of the submitted clinical data, and no judgment is made in the letter on the efficacy or safety of Firdapse.
Largely an administrative folly, an article on The Street referred to the letter as both "rare and embarrassing" for Catalyst.
Read the Catalyst press release