The US FDA has approved cannabidiol (Epidiolex) for the treatment of seizures associated with tuberous sclerosis complex (TSC) in patients one year of age and older.
Cannabidiol (CBD) is an oral drug of the Cannabis sativa plant. However, CBD does not cause intoxication or euphoria that comes from tetrahydrocannabinol (THC). CBD 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).
TSC is a rare genetic disease that causes non-cancerous (benign) tumours 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 behavioural problems, although the signs and symptoms of the condition, as well as the severity of symptoms, vary widely.
The approval for CBD in the treatment of seizures associated with TSC was provided based on a randomized, double-blind, placebo-controlled trial where 148 patients out of a total of 224 in the study received the drug.
Patients treated with CBD 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, said the researchers.
The most common side effects that occurred in CBD-treated patients with TSC in the clinical trial included diarrhoea, elevated liver enzymes, decreased appetite, sleepiness, fever, and vomiting.