Catalyst Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted the company orphan drug designation for Firdapse® (amifampridine phosphate) for the treatment of myasthenia gravis.
Myasthenia Gravis caused by antibodies to the muscle-specific kinase (MuSK-MG) is a rare disease that is estimated to inflict 5-8% of all myasthenia gravis patients (equating to an estimate of approximately 4,500 patients in the United States). MuSK antibodies identify a clinically distinguishable, more severe form of MG. The disease is characterized by a predominance in females, prominent bulbar involvement, more severe clinical condition and resistance to treatment. Although many patients with MuSK-MG are presently treated with anticholinesterase inhibitors or immunosuppressants, such patients do not generally respond adequately to these treatments.
"We are pleased that the FDA has granted Orphan Drug designation to Firdapse for myasthenia gravis, as it provides Catalyst with a number of benefits through development and commercialization," stated Patrick J. McEnany, Chief Executive Officer of Catalyst. He continued, "We are currently supporting an investigator-sponsored, randomized, double-blind, placebo controlled study evaluating Firdapse for the treatment of patients with MuSK-MG, and we anticipate the investigator reporting top-line results from this study in early 2017. If this trial is successful, and subject to the availability of funding, we hope to initiate a registration quality trial in the U.S. evaluating Firdapse for the treatment of patients with MuSK-MG."
