In documents released Tuesday ahead of an FDA advisory committee meeting, agency staff said that GW Pharmaceuticals provided "substantial evidence" of the effectiveness of Epidiolex (cannabidiol) in treating seizures associated with Lennox-Gastaut syndrome and Dravet syndrome. "The risk-benefit profile established by the data in the application appears to support approval," the FDA reviewers noted. Shares in the drugmaker rose as much as 13.2 percent on the news.
GW's submission of Epidiolex is backed by efficacy and safety from three studies involving a total of more than 500 patients. In late-stage study data unveiled in 2016, the drug, when used in combination with current anti-epileptic medication, was associated with a significant decrease in seizure frequency versus anti-epileptic drug alone in patients with Lennox-Gastaut syndrome. Similar findings were also reported in a prior study of patients with Lennox-Gastaut syndrome as well as in patients with Dravet syndrome.
The advisory committee meeting will be held on April 19, with the FDA set to make a decision on whether or not to approve Epidiolex by June 27. If approved, the therapy would be the first drug derived directly from the cannabis plant to be cleared in the US. GW chief executive officer Justin Gover said "Epidiolex, if approved, will mark a sea change in the acceptability of cannabinoids as therapy."
Epidiolex was granted orphan drug status in 2014 by the FDA in the treatment of Lennox-Gastaut syndrome. Separately, the therapy was awarded fast track and orphan drug status by the drug regulator for use in patients with Dravet syndrome.
Epidiolex is also under review by the European Medicines Agency for the treatment of both Dravet syndrome and Lennox-Gastaut syndrome.
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