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.
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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.
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"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."
