BioMarin Pharmaceutical has licensed North American rights to its EC-approved orphan drug Firdapse™ to Catalyst Pharmaceutical Partners, and is making a $5 million investment in Catalyst to support the latter’s development of the drug in the U.S. Firdapse (amifampridine phosphate) is a calcium channel blocker that is approved in the EU for treating Lambert-Eaton Myasthenic Syndrome (LEMS), and is currently undergoing Phase III LEMS trials in the U.S. LEMS is a sometimes fatal disease caused by autoantibodies to voltage-gated calcium channels, which causes muscle weakness symptoms, and can be life-threatening when the weakness involves respiratory muscles.

Under terms of the deal Catalyst has exclusive rights to Firdapse for all indications in North America, and will shoulder all future development and commercialization costs in its designated markets. The firm will share with BioMarin the costs of post-marketing studies in the EU, data from which are expected to be included in the registration dossier for Firdapse in the U.S. Subject to certain conditions BioMarin could also be due royalty payments.

“Our existing product candidates are focused on addiction and central nervous system orphan indications like infantile spasms/West syndrome and Tourette’s disorder, and adding Firdapse is consistent with our product development strategy,” states Patrick J. McEnany, Catalyst’s CEO. “The relationship with BioMarin is exciting and strategically important, as it provides Catalyst with another orphan drug candidate and near-term funding towards the completion of the currently underway Phase III trial for Firdapse.”

BioMarin acquired Firdapse when it took over Huxley Pharmaceuticals in 2009, and has brought the drug to market in the EU. Additional potential orphan CNS indications for the drug include myasthenia gravis and congenital myasthenic syndrome.

Catalyst is developing drugs against addiction, for pain management, and for treating CNS diseases including epilepsy. The firm’s lead products, CPP-109 and CPP-115, are vigabatrin analogues in various stages of clinical/preclincial development for treating addiction to cocaine, methamphetamine, opiates, and alcohol, and for treating epilepsy, respectively.

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