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news.UK-based GW Pharmaceuticals has received confirmation from the US Food and Drug Administration (FDA) that its investigational new drug (IND) application is now open for Epidiolex to treat Dravet Syndrome.
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Dravet Syndrome is also known as Severe Myoclonic Epilepsy of Infancy (SMEI), a rare and catastrophic treatment-resistant form of childhood epilepsy.
Following confirmation from FDA, the company intends to start a Phase II/III clinical trial in the second half of 2014.
Epidiolex has already been designated with orphan drug status by the US FDA for the treatment of Dravet syndrome.
GW chief executive officer Justin Gover said the acceptance of this IND by the FDA is a significant milestone for Epidiolex and for children with Dravet syndrome for whom existing anti-epileptic medicines often do not provide adequate relief.
"This journey began with requests from the U.S. epilepsy physician and patient community to utilize Epidiolex and has now led to GW embarking on its own formal development program with a view to seeking market authorization from the FDA as rapidly as possible," Gover said.
"We are committed to providing a prescription cannabidiol (CBD) medicine for children with Dravet syndrome who have exhausted all other therapeutic options."
The Phase II/III trial is a two-part randomized double-blind, placebo-controlled parallel group dose escalation, safety, tolerability, pharmacokinetic and efficacy study.
The trial is designed to assess single and multiple doses of Epidiolex in the treatment of Dravet syndrome in children who are being treated with other anti-epileptic drugs.
According to the company, first part of the trial includes the pharmacokinetic and dose-finding elements in a total of 30 patients over a three-week treatment period, while the part two is a placebo-controlled safety and efficacy evaluation of the drug over a three-month treatment period in a total of 80 patients.
The company also plans to initiate additional Phase III trial in Dravet syndrome in the first quarter of 2015 in parallel with part two of the first Phase II/III trial.
Image: GW’s Phase 2/3 trial will evaluate multiple doses of Epidiolex in the treatment of Dravet syndrome in children. Photo: courtesy of GW Pharmaceuticals 2014