Catalyst Pharmaceuticals Announces a Clinical Trial for Pediatric Patients With Congenital Myasthenic Syndromes

Catalyst Pharmaceuticals, Inc. CPRX, a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating diseases, today announced a clinical trial with Firdapse® (amifampridine phosphate) in pediatric patients with congenital myasthenic syndromes (CMS). Firdapse has received Breakthrough Therapy Designation from the FDA for the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS), as well as orphan drug designations for LEMS and CMS. Patrick J. McEnany, Catalyst's Chief Executive Officer said, "We continue to work towards the completion of our rolling submission of the NDA for Firdapse, which, as previously announced, we expect to complete during the fourth quarter of 2015. Our initial NDA submission will include data and information on the benefits of Firdapse for certain types of CMS and will request that CMS be included in our label for Firdapse. Our initial NDA submission will also include the positive results seen to date in children with CMS who are currently being treated with Firdapse under an investigator treatment IND." Dr. Steven Miller, Catalyst's Chief Operating Officer and Chief Scientific Officer said, "With regard to our development plan for CMS, in addition to an investigator sponsored IND, we have initiated a small blinded clinical trial in the pediatric CMS population, ages 2 to 17. When the data from this new study is available, we intend to amend our NDA filing with these data. The design of this new study is based on the guidance that the FDA provided to us during our pre-NDA meeting in early 2015. Several academic institutions have been recruited for this new trial. We expect to complete this study by April of next year and at that time participants in this study may continue to receive Firdapse through our Expanded Access Program." Additional information about this trial (NCT02562066) can be found on www.clinicaltrials.gov. About Congenital Myasthenic Syndromes Congenital myasthenic syndromes, or CMS, is a rare neuromuscular disease comprising a spectrum of genetic defects and is characterized by fatigable weakness of skeletal muscles with onset at or shortly after birth or early childhood; in rare cases symptoms may not manifest themselves until later in childhood. The severity and course of the disease are variable, ranging from minor symptoms to progressive disabling weakness; symptoms may be mild, but sudden severe exacerbations of weakness or even sudden episodes of respiratory insufficiency also occur. Congenital myasthenic syndrome(s) is rare, estimated at one-tenth that of myasthenia gravis, which in itself is rare. Based on currently available information, we estimate that there are between 1,000 and 1,500 CMS patients in the United States.