The Food and Drug Administration granted orphan drug status to Firdapse, an experimental treatment for patients with myasthenia gravis, a degenerative neuromuscular disease similar to Parkinson's or Lou Gehrig's disease, according to South Florida Business Journal.
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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