Catalyst Pharmaceuticals begin testing its orphan drug on first patients

Catalyst Pharmaceuticals, a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating diseases, has announced that the first patient has been enrolled into its second Phase 3 clinical trial (designated as LMS-003) to evaluate the efficacy and safety of Firdapse (amifampridine phosphate) in patients with Lambert-Eaton myasthenic syndrome (LEMS).

In October 2016, Catalyst announced that it had reached an agreement with the U.S. Food and Drug Administration (FDA) under a Special Protocol Assessment (SPA) for the protocol design, clinical endpoints, and statistical analysis approach in this additional Phase 3 trial. Catalyst has previously announced positive results from its first Phase 3 clinical trial evaluating Firdapse for the treatment of LEMS.

“The initiation of our second Phase 3 study of Firdapse in LEMS patients is a significant step forward on our path towards gaining approval for Firdapse,” said Patrick J. McEnany, Catalyst’s Chief Executive Officer. “We believe we have a clearly defined development and regulatory pathway for Firdapse in the treatment of LEMS and remain focused on our goal of improving access to treatments for patients with this significant unmet need.”

“Based on the positive results seen in our previous Phase 3 study, we look forward to continuing to investigate Firdapse for the treatment of LEMS,” said Gary Ingenito, M.D., Ph.D., Chief Medical Officer of Catalyst. “We are grateful to the patients and families, as well as their physicians, who have participated in our expanded access program and are participating in this second Phase 3 study.”