- Catalyst Pharmaceuticals (CPRX +5.8%) initiates the rolling submission of its New Drug Application for Orphan Drug- and Breakthrough Therapy-tagged Firdapse (amifampridine) for the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS). The rolling submission, intended to shorten the regulatory review clock, is a benefit of BTD status.
- LEMS is a rare autoimmune disorder that is characterized by muscle weakness in the arms and legs caused by the buildup of antibodies at the connection between nerve and muscle (neuromuscular junction). It commonly strikes people over the age of 40 and is prevalent in those with lung cancer. It affects ~4,700 people in North America.
- The European Commission approved Firdapse for the treatment of LEMS in 2010. Catalyst acquired the North American rights to the product in October 2012 from developer BioMarin Pharmaceutical (BMRN +0.6%) as part of a strategic collaboration.
- Amifampridine enhances neuromuscular transmission by blocking the potassium channel. This causes depolarization of the presynaptic membranes which opens the calcium channel.
Catalyst Pharma starts rolling NDA submission for Firdapse in LEMS
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