Expects to Complete Full NDA Submission in the Fourth Quarter of 2015
CORAL GABLES, FL, USA I July 22, 2015 I Catalyst Pharmaceuticals, Inc. (Nasdaq:CPRX), a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating diseases, today announced the initiation of a rolling submission of a New Drug Application (NDA) to the United States (U.S.) Food and Drug Administration (FDA) for Firdapse® for the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS). Firdapse® has received Breakthrough Therapy Designation from the FDA for the treatment of LEMS, as well as orphan drug designations for LEMS and congenital myasthenic syndromes (CMS).
“Our start of the NDA submission for Firdapse® marks an important step forward in our efforts to provide a safe and effective, FDA approved, treatment option for patients in the U.S. who develop LEMS, a rare, debilitating disease,” said Patrick J. McEnany, Chief Executive Officer of Catalyst. “We expect to complete the submission of the NDA in the fourth quarter of 2015, at which time we will be requesting a Priority Review of our application. We will continue to work closely with the FDA as we seek approval of the NDA. As part of our commitment to ensure that eligible patients have access to Firdapse® as we pursue U.S. approval, we will continue to provide access to Firdapse® through an expanded access program.”
The Breakthrough Therapy Designation is designed to convey all of the fast track program features, as well as more intensive FDA guidance on an efficient drug development program. The Fast Track Designation is designed to facilitate the development and expedite the review of drugs that treat serious, life-threatening conditions and that address unmet medical needs. The Fast Track process allows a company to submit individual modules of its NDA for review by the FDA as they are completed.
AboutCatalyst 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, recently completed testing in a global, multi-center, pivotal Phase 3 trial resulting in positive top-line data. 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.
SOURCE: Catalyst Pharmaceuticals
Post Views: 91
Expects to Complete Full NDA Submission in the Fourth Quarter of 2015
CORAL GABLES, FL, USA I July 22, 2015 I Catalyst Pharmaceuticals, Inc. (Nasdaq:CPRX), a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating diseases, today announced the initiation of a rolling submission of a New Drug Application (NDA) to the United States (U.S.) Food and Drug Administration (FDA) for Firdapse® for the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS). Firdapse® has received Breakthrough Therapy Designation from the FDA for the treatment of LEMS, as well as orphan drug designations for LEMS and congenital myasthenic syndromes (CMS).
“Our start of the NDA submission for Firdapse® marks an important step forward in our efforts to provide a safe and effective, FDA approved, treatment option for patients in the U.S. who develop LEMS, a rare, debilitating disease,” said Patrick J. McEnany, Chief Executive Officer of Catalyst. “We expect to complete the submission of the NDA in the fourth quarter of 2015, at which time we will be requesting a Priority Review of our application. We will continue to work closely with the FDA as we seek approval of the NDA. As part of our commitment to ensure that eligible patients have access to Firdapse® as we pursue U.S. approval, we will continue to provide access to Firdapse® through an expanded access program.”
The Breakthrough Therapy Designation is designed to convey all of the fast track program features, as well as more intensive FDA guidance on an efficient drug development program. The Fast Track Designation is designed to facilitate the development and expedite the review of drugs that treat serious, life-threatening conditions and that address unmet medical needs. The Fast Track process allows a company to submit individual modules of its NDA for review by the FDA as they are completed.
AboutCatalyst 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, recently completed testing in a global, multi-center, pivotal Phase 3 trial resulting in positive top-line data. 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.
SOURCE: Catalyst Pharmaceuticals
Post Views: 91
