Epidiolex (Cannabidiol) Primer: Frequently Asked Questions for Patients and Caregivers

Abstract

On June 25, 2018, the first plant-derived, purified pharmaceutical-grade cannabidiol (CBD) medication, Epidiolex (Greenwich Biosciences Inc, Carlsbad, CA), was approved in the United States by the US Food and Drug Administration (FDA). Epidiolex is for patients 2 years and older with Dravet syndrome (DS) or Lennox-Gastaut syndrome (LGS), and has revolutionized the treatment of medically refractory seizures in these patients.1–4 The road to widespread therapeutic use of CBD and its recent approval for seizure disorders has been a long one.1–4Four pivotal randomized, double-blind, multicenter clinical trials evaluated the use of CBD in patients with DS and LGS with regard to the efficacy and safety in convulsive and drop seizure, respectively (GWPCARE 1–4).4 All trials demonstrated a significant absolute reduction in seizure frequency.1–4 The most common adverse drug effects (≥10%) depicted in the trials included somnolence, fatigue, rash, decreased appetite, diarrhea, insomnia, infection, and elevated transaminases.1–4Epidiolex is a CBD-only component, with no Δ9 tetrahydrocannabinol [Δ9 THC].1 THC is the psychoactive component of cannabis, responsible for appetite stimulation and a euphoria sensation.56 When referring to cannabis it is a collective term for 3 species of hemp plants, including Cannabis sativa, Cannabis indica, and Cannabis ruderalis.56 Both THC and CBD are considered phytocannabinoids, which are chemicals within cannabis that may interact with the cannabinoid receptor.56 Although the exact mechanism of action is unknown, both CBD and THC are thought to be efficacious in their role for pediatric epilepsy.56 Using the non-psychoactive component, CBD, allows patients to have few side psychoactive side effects while still having the benefit of reduction in seizure frequency.6 There are other cannabidiol products currently on the market, including hemp oil. Hemp oil is federally legal and contains less than or equal to 0.3% THC and is hemp derived. It can be bought over the counter at pharmacies, grocery stores, and even gas stations.7 Some states allow patients or caregivers to possess CBD products greater than 5% with a state permit.7 Although it is becoming increasingly common to see patients on hemp oil because of the ease of access, it is important to recognize that none of these products are FDA approved and have efficacy data in relation to seizures.7 The FDA has released a statement saying it is “aware that some companies are marketing products containing cannabis and cannabis-derived compounds in ways that violate the Federal Food, Drug and Cosmetic Act (FD&C Act) and that may put the health and safety of consumers at risk.”8 The Table below contains a list of frequently asked questions regarding Epidiolex.