British biopharmaceutical firm GW Pharmaceuticals has received confirmation from the US Food and Drug Administration (FDA) that its investigational new drug application (IND) is now open for Epidiolex in the treatment of Dravet dyndrome, also known as severe myoclonic epilepsy of infancy (SMEI), a rare and catastrophic treatment-resistant form of childhood epilepsy.
Following confirmation from the FDA, the company intends to initiate a Phase II/III clinical trial in the second half of this year.
The drug has already been given designated with orphan drug status by the FDA for treatment of Dravet syndrome.
GW chief executive officer Justin Gover said 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 US epilepsy physician and patient community to utilise Epidiolex and has now led to GW embarking on its own formal development programme with a view to seeking market authorisation 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 two-part randomised double-blind, placebo-controlled parallel group dose escalation, safety, tolerability, pharmacokinetic and efficacy Phase II/III trial will evaluate single and multiple doses of Epidiolex to treat Dravet syndrome in children who are being treated with other anti-epileptic drugs.
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.
According to the company, all patients who participate in the trial will be eligible to receive Epidiolex under a long term open label extension protocol.
The company intends to start an 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.
As well as Dravet syndrome, the company plans to carry out a clinical development programme for Epidiolex in the treatment of Lennox-Gastaut syndrome (LGS).
Dravet syndrome is a rare and catastrophic treatment-resistant form of childhood epilepsy for which there is currently no cure.