Catalyst heads back to the FDA in search of an elusive OK after Firdapse scores high in new PhIII

About 18 months after the FDA thumbed Catalyst Pharmaceuticals $CPRX back to the clinic with a demand for more clinical data on its lead drug Firdapse (amifampridine phosphate), the biotech says that the therapy passed a second Phase III trial with flying colors. And that success paves the way to a new drug application which the company plans to deliver to the FDA in the next few months.

Patrick McEnany

Researchers recruited 26 patients for their latest Phase III, which studied the drug’s effect on symptoms of the ultra rare neuromuscular disease Lambert-Eaton myasthenic syndrome. According to Coral Gables, FL-based Catalyst, Firdapse hit with statistically significant results for both co-primary endpoints: The quantitative myasthenia gravis score (with a p-value of 0.0004) and subject global impression scores (with a p-value of 0.0003).

“Data from the LMS-003 trial continue to demonstrate that amifampridine phosphate has a significant magnitude of effect in treating the symptoms of LEMS patients,” said Perry Shieh, principal investigator of this clinical trial. “The findings are especially meaningful given the need for FDA-approved therapies which may transform the lives of patients suffering from LEMS.”

The FDA had sent the company back to the drawing board with a demand for more data after regulators concluded that one of its late-stage studies was incomplete. The FDA handed Catalyst a breakthrough drug designation on the drug back in 2013.

“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 Catalyst CEO Patrick McEnany.

The best place to read Endpoints News? In your inbox.

Comprehensive daily news report for those who discover, develop, and market drugs. Join 51,200+ biopharma pros who read Endpoints News by email every day.

Free Subscription

VP Oncology Biology
Skyhawk Therapeutics Waltham, MA
Associate Director CMC
Elektroki Boston, MA
Director Process Development
Elektroki Boston, MA
Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT

Visit Endpoints Careers ->