EPIDYOLEX

What it is

EPIDYOLEX is a cannabidiol oral solution containing 100 mg of cannabidiol per mL. This medicinal product is subject to additional monitoring in Australia to allow quick identification of new safety information.

Approved indications —

Adjunctive therapy of seizures associated with Lennox-Gastaut syndrome for patients 2 years of age and older — Adjunctive therapy of seizures associated with Dravet syndrome for patients 2 years of age and older

Dosing overview

The recommended starting dose is 2.5 mg/kg taken twice daily (5 mg/kg/day) for one week. After one week, the dose should be increased to a maintenance dose of 5 mg/kg twice daily (10 mg/kg/day). Based on individual clinical response and tolerability, each dose can be further increased in weekly increments of 2.5 mg/kg administered twice daily (5 mg/kg/day) up to a maximum recommended dose of 10 mg/kg twice daily (20 mg/kg/day). EPIDYOLEX should be initiated and supervised by a neurologist. Food may increase EPIDYOLEX levels and therefore it should be taken consistently either with or without food, including the ketogenic diet. When taken with food, a similar composition of food should be considered, if possible.

Key safety warnings

EPIDYOLEX can cause dose-related elevations of liver transaminases (alanine aminotransferase and/or aspartate aminotransferase). The elevations typically occur in the first two months of treatment initiation; however, there were cases observed up to 18 months after initiation of treatment, particularly in patients taking concomitant valproate. Transaminase elevations of greater than 3 times the upper limit of normal in the presence of elevated bilirubin without an alternative explanation are an important predictor of severe liver injury. Early identification of elevated transaminase may decrease the risk of a serious outcome. Prior to starting treatment with EPIDYOLEX, obtain serum transaminases (ALT and AST) and total bilirubin levels. Serum transaminases and total bilirubin levels should be obtained at 1 month, 3 months, and 6 months after initiation of treatment with EPIDYOLEX, and periodically thereafter or as clinically indicated. EPIDYOLEX can cause somnolence and sedation, which occur more commonly early in treatment and may diminish with continued treatment. The occurrence was higher for those patients on concomitant clobazam. Other CNS depressants, including alcohol, can potentiate the somnolence and sedation effect. EPIDYOLEX should generally be withdrawn gradually because of the risk of increased seizure frequency and status epilepticus. If withdrawal is needed because of a serious adverse event, rapid discontinuation can be considered. Suicidal behaviour and ideation have been reported in patients treated with AEDs in several indications. A meta-analysis of randomised placebo-controlled trials with AEDs has shown a small increased risk of suicidal behaviour and ideation. The mechanism of this risk is not known, and the available data do not exclude the possibility of an increased risk for EPIDYOLEX. Patients should be monitored for signs of suicidal behaviour and ideation and appropriate treatment should be considered.

Contraindications

Hypersensitivity to the active substance or to any of the excipients listed in section 6.1. Patients with transaminase elevations greater than 3 times the upper limit of normal and bilirubin greater than 2 times the upper limit of normal.

PBS listing

EPIDYOLEX oral liquid 100 mg per mL is listed on the PBS in a 100 mL bottle formulation with 2 PBS items. The restriction type is authority required (streamlined), and the ex-manufacturer price is A$1400.00.

Regulatory history

EPIDYOLEX cannabidiol 100 mg/mL oral liquid solution was first listed on the ARTG on 21 September 2020. The TGA approved Epidyolex for adjunctive therapy of seizures associated with Lennox-Gastaut syndrome or Dravet syndrome in patients 2 years and older following an assessment on 18 September 2020. In November 2020, PBAC recommended the medicine for Dravet syndrome but did not recommend it for Lennox-Gastaut syndrome. In July 2025, PBAC recommended the medicine for seizures associated with Lennox-Gastaut syndrome and recommended amending the restriction level to authority required (streamlined) to ease clinical criteria and improve access.