The US Food and Drug Administration (FDA) has issued Breakthrough Therapy Designation for Catalyst Pharmaceutical Partners’ Firdapse (amifampridine phosphate), which is used to treat patients with Lambert-Eaton Myasthenic Syndrome (LEMS).
Depending on the clinical data from several previously published clinical trials of amifampridine (3,4-DAP) in patients with LEMS, the FDA awarded the designation for Firdapse.
LEMS is an autoimmune disease that can be severely disabling, with muscle weakness as the primary symptom.
Firdapse is Catalyst's investigational therapy developed for the treatment of the LEMS symptoms, including muscle weakness.
Known as amifampridine phosphate, 3,4-diaminopyridine phosphate or 3,4-DAP phosphate, Firdapse is a potassium channel blocker that delays repolarization of the pre-synaptic neuron, causing voltage gated Ca2+ channels to remain open longer.
It offers relief to the LEMS symptoms, including muscle weakness, difficulty swallowing and talking, drooping of eyelids and facial weakness.
Catalyst Pharmaceutical Partners president and chief executive officer Patrick McEnany said, "With no approved or effective symptomatic treatment currently available for LEMS, Firdapse has the potential to be the first-line treatment option for patients with this rare condition."
