Catalyst Pharmaceuticals, Inc., a biopharmaceutical company focused on developing and commercialising innovative therapies for people with rare debilitating diseases, has begun rolling submission of a New Drug Application (NDA) to the United States 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 US 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 US approval, we will continue to provide access to Firdapse through an expanded access programme."
The Breakthrough Therapy designation is designed to convey all of the fast track programme features, as well as more intensive FDA guidance on an efficient drug development programme. 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.