GW Pharmaceuticals announced Monday that a Phase III study of the experimental drug Epidiolex (cannabidiol) for the treatment of Dravet syndrome met its primary endpoint, demonstrating a significant reduction in convulsive seizures compared with placebo. CEO Justin Gover said "in light of this positive data, we will now request a pre-NDA meeting with the FDA to discuss our proposed regulatory submission." Shares in the company rose as much as 140 percent on the news.
In the study, 120 patients with Dravet syndrome were randomised to treatment with Epidiolex or placebo, both in combination with anti-epileptic drug (AED) treatment regimens, for 14 weeks. GW Pharmaceuticals noted that patients were taking an average of three AEDs and previously failed an average of more than four AEDs. The main goal of the study was the percentage change in the monthly frequency of convulsive seizures during the treatment period versus the four-week baseline observation period.
Results showed that Epidiolex was associated with a 39 percent median reduction in monthly convulsive seizures, versus 13 percent for placebo. The company indicated that the difference became apparent in the first month of treatment and persisted throughout the remainder of the treatment period. The drugmaker added the significance of the finding was confirmed by a series of sensitivity analyses.
GW Pharmaceuticals explained that data from the secondary endpoints supported the overall efficacy of the therapy. The company plans to submit additional data from the study for publication and presentation at scientific meetings. "This shows that cannabinoids can produce compelling and clinical important data and represent a highly promising new class of medications, hopefully in a range of conditions," Gover remarked. Epidiolex is a liquid formulation of pure plant-derived cannabidiol.
GW Pharmaceuticals said that it is currently conducting a second Phase III study of Epidiolex in Dravet syndrome with a planned recruitment of 150 patients. The therapy was granted orphan drug designation for this indication by the FDA in 2013 and awarded fast track status the following year. The company has also initiated late-stage studies of Epidiolex for the treatment of Lennox-Gastaut syndrome, for which it has received FDA orphan drug status, as well as tuberous sclerosis complex. Analysts predict that if approved, the drug could generate sales of $1.1 billion by 2021.
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