Catalyst Pharmaceuticals

Catalyst Pharmaceuticals is a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating diseases, including Lambert-Eaton myasthenic syndrome (LEMS), congenital myasthenic syndromes (CMS), infantile spasms, and Tourette's Disorder. Catalyst's lead candidate, Firdapse® for the treatment of LEMS, has completed testing in a global, multi-center, double-blinded randomized pivotal Phase 3 trial resulting in positive top-line data and Catalyst has recently filed an NDA for this product seeking approval for its use as a treatment of LEMS and CMS. Firdapse® for the treatment of LEMS has received Breakthrough Therapy Designation from the U. S. Food and Drug Administration (FDA) and orphan drug designation for LEMS and CMS. Firdapse® is the first and only European approved drug for symptomatic treatment in adults with LEMS. Catalyst is also developing CPP-115 to treat infantile spasms, epilepsy and other neurological conditions associated with reduced GABAergic signaling, like post-traumatic stress disorder and Tourette's Disorder. CPP-115 has been granted U. S. orphan drug designation for the treatment of infantile spasms by the FDA and has been granted E. U. orphan medicinal product designation for the treatment of West Syndrome by the European Commission. In addition, Catalyst is developing a generic version of Sabril® (vigabatrin).