The Food and Drug Administration (FDA) has granted orphan drug designation to Epidiolex, an experimental medication that contains plant-derived Cannabidiol (CBD) for use in treating children with Dravet syndrome, a rare form of infant-onset, genetic, drug-resistant epilepsy.
Epidiolex is an oral liquid that contains as its active ingredient a highly purified extract of CBD, a non-psychoactive molecule of the cannabis plant, also known as marijuana.
Dravet syndrome is a rare epilepsy syndrome with a distinctive yet complex electroclinical presentation. It most often occurs during the first year of life with clonic and tonic-clonic seizures in previously healthy and developmentally normal infants. Prognosis, however, is poor and those with the condition often develop intellectual disabilities and life-long ongoing seizures.
There are an estimated 5,440 people living with Dravet syndrome in the United States and another 6,710 in Europe, however the condition may be under-diagnosed.
Orphan drugs are ones that are developed specifically to treat rare medical conditions, which are conditions that are often referred to as orphan diseases. There are financial incentives, such as extended exclusivity periods, in the United States and Europe for drug companies to develop drugs to treat orphan diseases that otherwise lack a sufficient profit motive.
GW Pharmaceuticals, the company manufacturing Epidiolex, anticipates a meeting in the near future with the FDA to discuss a development plan for its drug. The company has also made arrangements to enable independent U.S. pediatric epilepsy specialists to treat high-need pediatric epilepsy cases with Epidiolex immediately.