A Food and Drug Administration advisory panel has unanimously recommended approval of a new epilepsy medication made with an ingredient found in marijuana. The drug, called Epidiolex, is made by GW Pharmaceuticals, a British company. If the agency follows the recommendation, the drug would be the first plant-derived cannabidiol prescription medicine available in the United States. The F.D.A. is not bound by advisory committee recommendations but often follows them.
The panel recommended approval of the drug to treat two rare forms of epilepsy that are among the most difficult types of epilepsy to treat — Lennox-Gastaut syndrome and Dravet syndrome. There are an estimated 30,000 children and adults with Lennox-Gastaut syndrome, a type of epilepsy with multiple types of seizures. Lennox-Gastaut syndrome usually appears between ages 3 and 5. Dravet syndrome is a rare genetic dysfunction of the brain that begins in the first year of life.
The recommendation noted that GW had submitted positive results of efficacy from three randomized, double-blind, placebo-controlled trials conducted in patients with both diseases. Nearly all patients with these conditions continue to have seizures despite currently available medications. The briefing materials prepared for the committee said, “The statistically significant and clinically meaningful results from these three studies provide substantial evidence of the effectiveness of CBD for the treatment of seizures associated with LGS and DS.”
GW Pharmaceuticals has received an orphan drug designation for Epidiolex, reserved for drugs that are developed to treat rare conditions. GW CEO Justin Gover said, “We’re obviously very pleased by the unanimous recommendation in support of the approval of Epidiolex. It’s a very important milestone in the approval process.”
Epidiolex, an oral solution, would be the first of a new class of drugs to treat epilepsy. The drug’s active ingredient, cannabidiol, also called CBD, is one of the chemical compounds found in the cannabis plant. However, the medication does not make people high. The FDA will vote in June whether to approve the drug.
According to the Epilepsy Foundation, one-third of Americans who have epilepsy have found no therapies that will control their seizures, affecting about 1 million families across the nation. Seizures put children at high risk for intellectual and developmental disabilities, and may result in death.