Catalyst Pharmaceuticals Completes NDA Submission to FDA for Firdapse for the Treatment of Lambert-Eaton Myasthenic Syndrome and Congenital Myasthenic Syndromes

CORAL GABLES, Fla., Dec. 17, 2015 (GLOBE NEWSWIRE) — Catalyst Pharmaceuticals, Inc. (Nasdaq:CPRX), a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating diseases, announced today the completion of its rolling submission of a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) for Firdapse® (amifampridine phosphate) for the treatment of Lambert Eaton myasthenic syndrome (LEMS) and congenital myasthenic syndromes (CMS). Both of these diseases are ultra-rare, with prevalences in the United States believed to be approximately 3,000 and 1,000-1,500 patients, respectively.“The completion of our NDA submission for Firdapse represents the culmination of years of effort by our employees, investigators, clinical trial sites, and most importantly the patients and families of the LEMS and CMS communities,” said Patrick J. McEnany, Chief Executive Officer of Catalyst. “Our NDA submission includes more than 60 pre-clinical and clinical studies conducted over the past 5 years by both Catalyst and BioMarin Pharmaceutical. The submission of this NDA brings us one step closer to making a treatment for LEMS and CMS available to all patients, not just a selected few fortunate enough to be treated by physicians holding INDs to treat these diseases with experimental variations of 3,4-DAP. We look forward to working with the FDA during the regulatory process in pursuit of our goal of bringing Firdapse to patients suffering with LEMS and CMS.”The NDA submission includes a request for a Priority Review by the FDA. Previously, Firdapse has received Breakthrough Therapy designation from the FDA for the treatment of LEMS, as well as Orphan Drug designations for LEMS and CMS.Shin Oh, M.D., Distinguished Professor Emeritus in the Department of Neurology at the University of Alabama at Birmingham School of Medicine, said, “As a clinician who treats patients with LEMS and CMS, there is a great deal of value in having an FDA approved drug that would include approved labeling and prescribing information, as well as pharmacovigilance. If Firdapse is approved by the FDA, patients currently without any access to effective drugs, which make up the vast majority, will be able to receive a treatment that has been shown to be both safe and highly effective.”About Lambert-Eaton Myasthenic Syndrome