Catalyst Pharmaceuticals, Inc., a biopharmaceutical company focused on developing and commercializing  therapies for people with rare diseases, has produced positive top-line results from a second Phase 3 clinical trial of Firdapse. The drug is used for the symptomatic treatment of Lambert-Eaton myasthenic syndrome (LEMS).  
 

This clinical trial was conducted using a protocol agreed to by the FDA through the Special Protocol Assessment (SPA) process, and had two prospectively defined co-primary endpoints. The first of these, quantitative myasthenia gravis score (QMG), achieved a statistically significant p-value of 0.0004, and the second, subject global impression (SGI), achieved a statistically significant p-value of 0.0003.

“We are extremely pleased with the top-line efficacy and safety results from this second Phase 3 trial, which reinforces the potential of Firdapse to be an important treatment for patients suffering from LEMS. We look forward to presenting further data in future publications and at medical conferences. We remain on track to submit our NDA to the FDA in the first quarter of 2018,” said Patrick J. McEnany, president and chief executive officer of Catalyst Pharmaceuticals.
 

This successful report comes after Firdapse first received Breakthrough Therapy designation from the FDA in August 2013.