The US Food and Drug Administration (FDA) has approved the cannabidiol (CBD) drug Epidiolex oral solution for the treatment of seizures associated with tuberous sclerosis complex (TSC) in patients one year of age and older, according to press release from GW Pharma.
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. TSC affects about 1 in 6,000 people, according to the release.
Epidiolex 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.
Epidiolex’s effectiveness for TSC-associated seizures was established in a randomized, double-blind, placebo-controlled trial where 148 patients out of 224 received Epidiolex. Patients treated with Epidiolex had a significantly greater reduction in the frequency of seizures during the 16-week treatment period than patients who received placebo.
The most common side effects in Epidiolex-treated patients were: diarrhea, elevated liver enzymes, decreased appetite, sleepiness, fever, and vomiting. The FDA had granted Priority Review designation for this application.