Firdapse amifampridine regulatory update

FDA granted breakthrough therapy designation to Catalyst's Firdapse amifampridine to treat Lambert-Eaton myasthenic syndrome (LEMS), a rare autoimmune disorder characterized by muscle weakness caused by impaired presynaptic release of acetylcholine. The potassium channel blocker is in Phase III testing in the U.S., with data expected in 2Q14 and an NDA submission planned for late 2014 or early 2015. Firdapse is already approved for LEMS in the EU, where it has Orphan Drug status. It also has Orphan Drug designation in the U.S. and Switzerland.

Last year, BioMarin granted Catalyst exclusive, North American rights to Firdapse (see BioCentury, Nov. 5, 2012). BioMarin gained the compound through its 2009 acquisition of Huxley Pharmaceuticals Inc. which licensed it from EUSA Pharma Inc. Jazz acquired EUSA last year (see BioCentury, Nov. 2, 2009 & June 18, 2012). ...