Catalyst Pharmaceuticals, Inc. (NASDAQ:CPRX), a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating diseases, announced today that it has reached an agreement with the U.S. Food and Drug Administration (FDA) under a Special Protocol Assessment (SPA) for the protocol design, clinical endpoints, and statistical analysis approach to be taken in Catalyst’s upcoming Phase 3 study evaluating Firdapse® (amifampridine phosphate) for the symptomatic treatment of Lambert-Eaton myasthenic syndrome (LEMS).
A SPA is a process by which sponsors ask the FDA to evaluate the protocol of a proposed clinical trial to determine whether it adequately addresses scientific and regulatory requirements for the purpose identified by the sponsor. A SPA agreement indicates concurrence with the adequacy and acceptability of specific critical elements of protocol design, endpoints and analysis. Additionally, it provides a binding agreement with FDA’s review division that a pivotal trial design, conduct, and planned analysis adequately addresses the scientific and regulatory objectives in support of a regulatory submission for drug approval. However, final marketing approval depends upon the results of efficacy, the safety profile, and an evaluation of the risk/benefit of treatment demonstrated in the Phase 3 clinical trial, among other requirements. For further information regarding the SPA process, please visit the FDA website at www.fda.gov.
“Receipt of this SPA agreement is a major milestone which provides us with a clearly defined development and regulatory pathway for Firdapse in the treatment of LEMS”, said Patrick J. McEnany, Catalyst’s Chief Executive Officer. “We would like to thank the FDA for its engagement and guidance in this process.”
Catalyst intends to conduct its second Phase 3 trial (designated as LMS-003) at two clinical trial sites, one on the east coast of the United States and one on the west coast of the United States. This double-blind, placebo controlled withdrawal trial will include approximately 28 subjects, so that the trial is adequately powered, and will have the same co-primary endpoints as Catalyst’s first Phase 3 trial evaluating Firdapse for the treatment of LEMS. Further, the FDA has agreed to allow Catalyst to enroll patients from its expanded access program as study subjects in this second trial. Finally, after further discussion with the FDA in connection with the SPA request, this second trial will be a parallel design and not a cross-over design.
Final details of the Phase 3 clinical trial will be available at the launch of the study on www.clinicaltrials.gov. As previously reported, Catalyst expects to initiate this trial before the end of this year.
About 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. 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, CMS and Myasthenia Gravis. Firdapse is the first and only approved drug in Europe 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).
