The FDA has approved a new indication for medicines containing active substances derived from cannabis for for treating seizures in rare genetic diseases. On 31 July 2020, the US Food and Drug Administration approved Epidiolex oral (Cannabidiol) [CBD] solution for seizures associated with tuberous sclerosis complex (TSC), for the treatment of patients aged one year and older. Epidiolex has previously been approved for seizures, associated with two rare and severe cases of epilepsy, Lennox-Gastaut syndrome (LGS) and Dravet Syndrome (DS). It is the only FDA-approved drug that contains purified medicinal substances derived from Cannabis. It is also the second FDA-approved drug to treat TSC-related seizures.
CBD is a chemical component of the Cannabis sativa plant
However, CBD does not cause intoxication or the euphoria ('high') that results from tetrahydrocannabinol
(THC). It is THC (not CBD) that is the main psychoactive component of Cannabis.
"The FDA continues to believe that the drug approval process is the best way to approve new medicines,
including all cannabis-derived medicines, to be made available to patients in need of appropriate
medical therapies, such as the treatment of seizures associated with these rare diseases. This paradigm
ensuring that new therapies are safe, effective and manufactured to a high quality, by ensuring
uniform and reliable dosing for patients," said Douglas Throckmorton, M.D., FDA Drug
Deputy Director of Regulatory Programmes at the Centre for Evaluation and Research. "The Agency is
Committed to supporting rigorous research into the potential uses of hemp-derived products
in medicine and to collaborate with product developers who have an interest in providing patients with safe and
effective, high-quality products."
TSC is a rare genetic disease that causes damage to the brain and other parts of the body such as the eyes, heart and kidneys,
lungs and skin, and the growth of cancerous (benign) tumours. TSC usually affects the central nervous system and can
cause a combination of symptoms including seizures, developmental delay and behavioural problems, although the condition
Signs and symptoms, as well as the severity of symptoms, vary widely. TSC affects approximately 1 in 6000
The efficacy of Epidiolex in the treatment of seizures associated with TSC was established in a randomised trial,
in a double-blind, placebo-controlled study in which Epidiolex was used by 148 of 224 patients
study participants. The study measured the change in seizure frequency from baseline. The study
patients treated with Epidiolex had a significantly lower seizure frequency during the treatment period
than patients receiving placebo (inactive treatment). This effect was observed during
eight weeks and remained constant throughout the 16-week treatment period.
Most common side effects.
The incidence of Epidiolex in TSC patients treated with Epidiolex prior to the clinical trial was:
diarrhoea, increased liver enzymes, decreased appetite, drowsiness, fever and
Vomiting. Additional side effects in patients with LGS, DS or TSC include: liver
irregularity, weight loss, anaemia and increased creatinine levels.
Epidiolex must not be given without a patient treatment guide describing important information about
the use and risks of the medicine. As with all medicines that currently treat epilepsy, including Epidiolex,
the most serious risks may be suicidal thoughts and an increase in behaviour, or thoughts about self
mutilation. Patients, their carers and their families should be advised to monitor for unusual
changes in mood or behaviour, such as worsening depression, suicidal thoughts or behaviour.
Patients, carers and family members should immediately report worrying behaviour to the Health
for care providers. Epidiolex has also caused liver damage in some patients.
Most cases have generally been mild, but there is a risk of rare but more severe liver damage. See
more severe liver damage may cause nausea, vomiting, abdominal pain and fatigue,
The FDA has given this programme a Priority Review designation. "Epidiolex approval has been granted
"Greenwich Biosciences Inc." of Carlsbad, California.
FDA, an agency of the US Department of Health and Human Services, protects public health
ensuring the availability of human and veterinary medicinal products, vaccines and other biological products for human use and
the safety, efficacy and security of medical devices. The Agency is also responsible for the US
safety of the country's food, cosmetics, food supplements and products emitting electromagnetic radiation, and
safety and regulation of tobacco products.