FDA approves Epidyolex for treatment of tuberous sclerosis complex-associated seizures

The US Food and Drug Administration (FDA) has approved Epidyolex (cannabidiol (CBD)) oral solution for the treatment of seizures associated with tuberous sclerosis complex (TSC) in patients one year of age and older. The approval was granted to Greenwich Biosciences Inc.

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

“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. This paradigm ensures new therapies are safe, effective and manufactured to a high quality that provides uniform and reliable dosing for patients,” said Dr Douglas Throckmorton, deputy centre director for regulatory programmes 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.”

According to the FDA, Epidyolex’s effectiveness for the treatment of seizures associated with TSC was established in a randomised, double-blind, placebo-controlled trial where 148 patients out of a total of 224 in the study received the drug. The study measured the change from baseline in seizure frequency. In the study, patients treated with Epidyolex 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.