Shares of Catalyst Pharmaceuticals were surging Friday morning after the biopharmaceutical firm said the Food and Drug Administration granted orphan drug status for Firdapse for the treatment of myasthenia gravis.
Myasthenia gravis is a rase disease caused by antibodies to the muscle-specific kinase (MuSK-MG) and is estimated to affect approximately 4,500 patients in the U.S. The company said that many patients with MuSK-MG are currently treated with anticholinesterase inhibitors or immunosuppressants, yet noted that such patients do not generally respond adequately to these treatments.
Catalyst Pharma said it is currently supporting a double-blind, placebo-controlled study evaluating Firdapse as a treatment for MuSK-MG patients and expects top-line results 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,” said Catalyst CEO Patrick McEnany.
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