The Orphan Drug Designation program offers various incentives for drugmakers to develop treatments for rare diseases that affect fewer than 200,000 Americans.
Firdapse is intended to treat about 5 percent of the myasthenia gravis patient population — around 4,500 people — who suffer from a specific type of the disease.
Catalyst Pharmaceuticals, located in Coral Gables, Fla., developed the drug. The company laid off a number of employees in May to fund additional trials of Firdapse.
Controlled trials of the drug are underway and Catalyst expects results in early 2017. The company will launch late-stage trials of the drug pending positive trial results and additional funding.
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