LONDON — The Food and Drug Administration has given orphan drug designation to an experimental treatment for a rare form of epilepsy.
GW Pharmaceuticals said Friday that the FDA had granted the designation to Epidiolex for Dravet syndrome, a severe, drug-resistant form of epilepsy that begins in infancy. The drug is an oral liquid of a highly purified extract of cannabidiol, a non-psychoactive molecule derived from the cannabis plant.
The FDA grants orphan drug designation to experimental treatments for diseases that affect fewer than 200,000 people in the United States. The agency has given physicians permission to use the drug to treat 125 children with Dravet syndrome. The disease affects about 5,440 people in the United States and causes lifelong, ongoing seizures and intellectual disability.