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CORAL GABLES, Fla., Oct. 29, 2013 (GLOBE NEWSWIRE) -- Catalyst Pharmaceutical Partners, Inc. (Nasdaq:CPRX), a specialty pharmaceutical company focused on developing safe and effective approved medicines targeting orphan neuromuscular and neurological diseases, provided an update on the status of its development activities for its lead product candidate, Firdapse™, which is being evaluated in a pivotal Phase 3 clinical trial for the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS).
The Phase 3 trial is designed as a randomized, double-blind, placebo-controlled discontinuation study followed by an open-label extension period in approximately 36-patients across 19 sites in the United States and Europe. Catalyst also expects to add approximately six sites in the United States (US), Canada, South America and Europe in the near future. Firdapse™ for the treatment of LEMS has recently been granted Breakthrough Therapy designation by the U.S. Food & Drug Administration (FDA).
Catalyst expects that it will report top-line results from the double-blind portion of this Phase 3 trial during the second quarter of 2014 and, if the trial results are successful, Catalyst expects to submit a new drug application (NDA) with the FDA in the first half of 2015. Further, as previously reported, an interim safety analysis was recently performed by the independent Data Safety Monitoring Committee that is part of the routine oversight of the Phase 3 trial. Following this meeting, the Committee recommended that Catalyst continue the trial as planned. This recommendation is based on the Committee's review of unblinded safety and clinical data available from the on-going trial.
Patrick McEnany, Catalyst's Chairman and CEO, commented, "Catalyst is dedicated to advancing important life-altering therapies targeting rare neuromuscular diseases. We are committed to combatting LEMS as our first orphan disease priority, followed by infantile spasms and Tourette syndrome. If we are successful in bringing Firdapse™ to market, it will be the first ever FDA approved product, demonstrating safety and efficacy for the treatment of LEMS. We expect that Catalyst and Firdapse™ will provide LEMS patients and their physicians with an alternative therapeutic option and easier access to on-going support."