Catalyst Pharmaceutical Partners has announced that Firdapse, the company’s investigational orphan drug for the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS), has been granted Breakthrough Therapy Designation by the FDA.
LEMS is a rare and debilitating autoimmune disease, and patients suffer from a variety of motor symptoms including muscle weakness, difficulty swallowing and talking, drooping of eyelids and facial weakness. The muscle weakness stems from autoantibodies generated against voltage-gated Ca2+ channels involved in the muscle action potential. Firdapse, a potassium channel blocker, holds voltage-gated Ca2+ channels open for longer and thus makes it more likely that a muscle action potential will be initiated.
The prevalence of LEMS in the US and Canada is estimated to be in the region of 3,000 patients, and no approved or effective symptomatic treatment exists in the US for the potentially life-threatening disease. Firdapse is currently marketed in the EU by BioMarin for the treatment of LEMS, and the North American rights to Firdapse were licensed to Catalyst in 2012. The drug is currently undergoing testing in a pivotal Phase III trial.
There have been a number of Breakthrough Therapy Designations awarded since the inception of the category last year. However, the list has mainly been dominated by large biopharmaceutical companies, so Catalyst’s addition is a rarity in itself.
The World Orphan Drug Congress Europe brings together pioneers from the orphan drug community, including key opinion leaders from the payer, HTA, public health and patient advocacy bodies. Read more about the World Orphan Drug Congress Europe 2013, 14-15 November 2013, Geneva.