Rare Disease Report

Cannabidiol Phase 3 Study Begins for Dravet Syndrome. NDA Submission Expected in 2016

MARCH 31, 2015
James Radke

A phase 3 study testing Epidiolex (cannabidiol) for the treatment of Dravet syndrome, a rare form of childhood epilepsy has begun. Top-line data is expected to be available by the end of the 2015.

Dravet syndrome, also known as severe myoclonic epilepsy of infancy, is a rare genetic epileptic encephalopathy that begins during the first year of life. The first seizure is often associated with a fever and may be a tonic clonic seizure or involve clonic movements on one side of the body. During the second year of life, other seizure types tend to manifest (eg, absence seizures, atonic seizures, partial seizures, nonconvulsive status epilepticus), and development often begins to decline.

The Phase 3 trial is a 14-week randomized double-blind, placebo-controlled parallel group safety, tolerability, pharmacokinetic and efficacy trial of cannabidiol to treat Dravet syndrome in children who are being treated with other anti-epileptic drugs. cannabidiol will be assessed as an adjunctive antiepileptic treatment. The primary measure of this trial will be the percentage change from baseline in convulsive seizure frequency during the maintenance period of the study in patients taking cannabidiol versus placebo. Several additional efficacy and safety secondary outcome measures will be analysed. Following their participation in the study, all patients will be eligible to receive cannabidiol under a long term open label extension study.

In a press release, Orrin Devinsky MD, Professor of Neurology, Neurosurgery, and Psychiatry at NYU School of Medicine, and Principal Investigator of the trial. Said:

"Dravet syndrome is a rare and catastrophic form of pediatric epilepsy in which there is a substantial unmet need and for which there are no specifically approved medicines in the United States."

"This Phase 3 clinical trial of Epidiolex in Dravet syndrome is the first ever rigorous placebo-controlled trial of pure pharmaceutical cannabidiol in the field of epilepsy and, as such, a trial of significant interest for the entire epilepsy physician and patient communities."

Justin Gover, Chief Executive Officer of GW Pharmaceuticals, the developer of the drug, added that a second Phase 3 trial is to begin shortly and the company plans to submit a New Drug Application for Epidiolex to the FDA in mid-2016.

GW anticipates commencing the second pivotal Phase 3 trial in Dravet syndrome soon and also expects to begin two Phase 3 clinical trials in patients with Lennox-Gastaut syndrome early in the second quarter of 2015. These additional pivotal trials are all expected to complete recruitment in 2015.

Lennox-Gastaut syndrome (is a severe childhood epileptic encephalopathy that is estimated to affect 15 per 100,000 persons. The syndrome is associated with multiple seizure types, including tonic, atonic, tonic clonic, myoclonic, and absence seizures.


GW Pharmaceuticals Initiates First Phase 3 Pivotal Trial for Epidiolex in Dravet Syndrome [press release]. London UK; March 31, 2015. https://globenewswire.com/news-release/2015/03/31/720523/10126878/en/GW-Pharmaceuticals-Initiates-First-Phase-3-Pivotal-Trial-for-Epidiolex-in-Dravet-Syndrome.html Accessed March 31, 2015.

Image of cannibas leaf courtesy wikimedia commons. https://commons.wikimedia.org/wiki/File:Cannabis_sativa_leaf_%281%29.jpg

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