Catalyst Pharmaceutical Partners, Inc. (CPRX) recently announced that Firdapse was given ‘Breakthrough Therapy’ designation by the U.S. Food and Drug Administration (:FDA).
The FDA gave the above-mentioned designation for the indication of Lambert-Eaton myasthenic syndrome (:LEMS).
We note that Catalyst Pharma is evaluating Firdapse, a phase III candidate, for the treatment of debilitating symptoms associated with LEMS, including muscle weakness.
The designation from the FDA was based on encouraging data from clinical trials of Firdapse in patients with LEMS.
We remind investors that Firdapse enjoys orphan drug status in the U.S. Catalyst Pharma acquired marketing rights to Firdapse from BioMarin Pharmaceutical (BMRN) in Oct 2012. Firdapse is currently in a phase III study for LEMS with top-line results from the double-blind portion of the study expected in the second quarter of 2014.
BioMarin Pharma currently markets Firdapse in the EU for the treatment of LEMS. Firdapse generated sales of $14.2 million in 2012, up 8.4% year over year.
Meanwhile, Catalyst Pharma also plans to evaluate Firdapse for the treatment of myasthenia gravis and congenital myasthenic syndrome, among others.
Apart from Firdapse, Catalyst Pharma is also looking to evaluate CPP-115 to treat epilepsy and other selected central nervous disease indications. We note that CPP-115 also enjoys orphan drug status in the U.S. for the treatment of infantile spasms and West’s syndrome (a form of infantile spasms) in the EU.
Catalyst Pharma is also looking to develop CPP-109 for the treatment of Tourette syndrome.
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