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Breakthrough Therapy Designation Granted for Catalyst Pharmaceutical Partners, Inc. (CPRX) Firdapse

Specialty pharmaceutical company Catalyst Pharmaceutical Partners announced that Firdapse (amifampridine phosphate), the company’s investigational product, has received “Breakthrough Therapy Designation” from the FDA for the symptomatic treatment of patients with Lambert-Eaton Myasthenic Syndrome (LEMS). Firdapse is an investigational therapy being evaluated for the treatment of the debilitating symptoms associated with LEMS, including muscle weakness.

Currently, there are no approved or effective symptomatic treatments for LEMS, which is a rare autoimmune disease, which gives Firdapse the potential to be the first-in-line treatment option for patients with this rare condition.

“We are very pleased to have received Breakthrough Therapy Designation for Firdapse, and we are excited by the FDA’s decision to place our product in a category that may enable expedited development and review for patients with LEMS,” said Catalyst President and CEO Patrick McEnany.

Firdapse’s receipt of Breakthrough Therapy Designation was based on clinical data from various previously published clinical trials of amifampridine (3,4-DAP) in LEMS patients. Firdapse can potentially offer great relief for the often debilitating symptoms of LEMS, which include muscle weakness, such as difficulty walking; difficulty swallowing and talking; eyelid drooping; and facial weakness.

Firdapse, which has previously received orphan drug designation, is currently undergoing testing in a global, multicenter, pivotal Phase III trial. In addition to LEMS, Firdapse has other potential orphan neuromuscular indications, including for myasthenia gravis and congenital myasthenic syndrome.

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