The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Catalyst Pharmaceuticals Inc. for Firdapse to treat myasthenia gravis (MG).
"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," Catalyst CEO Patrick J. McEnany said. "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.”
It is estimated that five percent to eight percent of patients with myasthenia gravis, which is approximately 4,500 patients in the United States, will be afflicted with the rare disease known as muscle-specific kinase (MuSK-MG), which causes MG to become more severe. Many of the patients who suffer with MuSK-MG are currently treated with anticholinesterase inhibitors or immunosuppressants, but do not respond well to this course of treatment.