Catalyst Pharmaceuticals files rolling submission NDA to FDA for Firdapse (amifampridine phosphate) to treat Lambert-Eaton Myasthenic Syndrome (LEMS).

Catalyst Pharmaceuticals announced the initiation of a rolling submission of a New Drug Application (NDA) to the FDA for Firdapse (amifampridine phosphate) for the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS). The company expects to complete the submission of the NDA in the fourth quarter of 2015, at which time it will be requesting a Priority Review of its application.

Comment:Lambert-Eaton Myasthenic Syndrome is a rare autoimmune disease that can be severely disabling, with the primary symptoms being severe muscle weakness. LEMS occurs when the immune system attacks the nerves that control the muscles. In LEMS, the body attacks nerve cell proteins that regulate the amount of acetylcholine (ACh) the nerves release. ACh is a neurotransmitter that controls muscle contractions such as walking, wiggling fingers, and shrugging of the shoulders. Specifically, the body attacks a protein called �voltage gated calcium channel� which is required for the release of ACh. When the nerves cannot release enough ACh -- because the voltage gated calcium channel is attacked -- the muscles are unable to work properly. The prevalence of LEMS is estimated at approximately 1 per 100,000 in the United States and Canada. Approximately 50% of people with LEMS also have cancer, most commonly small cell lung cancer (SCLC). In LEMS patients with SCLC, it is the cancer cells that induce the production of the antibodies, rather than a more general autoimmune state found in cancer-free LEMS patients.

Comment: Firdapse was EU approved in 2009.