The FDA has approved cannabidiol (CBD) (Epidiolex, Greenwich Biosciences Inc) oral solution for the treatment of seizures associated with tuberous sclerosis complex (TSC) for patients 1 year of age and older.
The indication is only the third approval for CBD, and follows the FDA’s recent draft guidance for conducting CBD research to facilitate and support safe and effective study results. The CBD treatment was previously approved for the treatment of seizures associated with 2 rare and severe forms of epilepsy, Lennox-Gastaut syndrome and Dravet syndrome. It is the second FDA approval of a drug for the treatment of seizures associated with TSC.
The approval for the CBD indication for TSC was based on a randomized, double-blind, placebo-controlled trial, which incorporated 224 participants receiving CBD.
CBD is an active chemical component of the cannabis plant; CBD is different from tetrahydrocannabinol (THC), which is the psychoactive component responsible for causing intoxication and euphoria that is often associated with cannabis.
TSC is a rare genetic disease, affecting an estimated 1 in 6000 people, that leads to benign tumors in the brain, eyes, heart, kidneys, lungs, and skin, often affecting the central nervous system. TSC can result in seizures, developmental delay, and behavioral problems; however, symptoms and severity can vary widely.
In the study in which CBD was evaluated for safety and efficacy, 148 patients from the 224 total participation population exhibited favorable results. Investigators assessed benefit through change in baseline in seizure frequency, where individuals receiving CBD showed significant improvements in seizure frequency amid the 16-week treatment period compared with placebo (inactive treatment).
Investigators reported diarrhea, elevated liver enzymes, decreased appetite, sleepiness, fever, and vomiting as the most common adverse effects for CBD treatment in this patient population.
The FDA news release additionally explained that administration of CBD must be offered in conjunction with a patient Medication Guide, which describes key information concerning the drug’s use and risks. According to the FDA, the most serious risks may include thoughts of suicide or self-harm.
The FDA granted Priority Review designation for the CBD application.
- FDA Approves New Indication for Drug Containing an Active Ingredient Derived from Cannabis to Treat Seizures in Rare Genetic Disease. News Release. FDA; July 31, 2020. Accessed August 4, 2020. https://www.fda.gov/news-events/press-announcements/fda-approves-new-indication-drug-containing-active-ingredient-derived-cannabis-treat-seizures-rare