FDA Approves Epidiolex to Treat Seizures in Rare Genetic Disease

Rare Daily Staff

The U.S. Food and Drug Administration approved GW Pharmaceuticals’ Epidiolex oral solution for the treatment of seizures associated with tuberous sclerosis complex in patients one year of age and older.

Epidiolex previously won approval for the treatment of seizures associated with two rare and severe forms of epilepsy, Lennox-Gastaut syndrome and Dravet syndrome. This is the only FDA-approved drug that contains CBD, a purified drug substance derived from cannabis. It is also the second FDA approval of a drug for the treatment of seizures associated with tuberous sclerosis complex (TSC).

TSC is a rare genetic disease that causes non-cancerous tumors to grow in the brain and other parts of the body like the eyes, heart, kidneys, lungs, and skin. It usually affects the central nervous system and can result in a combination of symptoms including seizures, developmental delay, and behavioral problems, although the signs and symptoms of the condition, as well as the severity of symptoms, vary widely. The condition affects about 1 in 6,000 people.

CBD is a chemical component of the Cannabis sativa plant. However, CBD does not cause intoxication or euphoria that comes from tetrahydrocannabinol (THC). It is THC (and not CBD) that is the primary psychoactive component of cannabis.

“The FDA continues to believe the drug approval process represents the best way to make new medicines, including any drugs derived from cannabis, available to patients in need of appropriate medical therapy such as the treatment of seizures associated with these rare conditions,” said Douglas Throckmorton, deputy center director for regulatory programs in the FDA’s Center for Drug Evaluation and Research. “The agency is committed to supporting rigorous scientific research on the potential medical uses of cannabis-derived products and working with product developers who are interested in bringing patients safe and effective, high quality products.”

The FDA based its approval on a randomized, double-blind, placebo-controlled trial where 148 patients out of a total of 224 in the study received Epidiolex. The study measured the change from baseline in seizure frequency. In the study, patients treated with Epidiolex had a significantly greater reduction in the frequency of seizures during the treatment period than patients who received placebo. This effect was seen within eight weeks and remained consistent throughout the 16-week treatment period.

The FDA granted Priority Review designation for this application. The agency granted approval of Epidiolex to Greenwich Biosciences, a subsidiary of U.K.-based GW Pharmaceuticals, which markets Epidiolex in the United States.