Epidiolex has both Orphan Drug Designation and Fast Track Designation from the U.S. Food and Drug Administration (FDA) in the treatment of Dravet syndrome, a rare and debilitating type of epilepsy for which there are now no treatments approved in the United States.
Advertisement
Top-line data from the first randomized controlled trial of cannabidiol (Epidiolex) indicated the drug was better than placebo at reducing seizures among children with Dravet syndrome, according to a press release from drugmaker GW Pharmaceuticals.
GW Pharma is already providing Epidiolex to more than 375 children with various forms of severe epilepsy under an expanded access program, which allows patients with serious conditions to use drugs that haven’t yet gained regulatory approval.
In this study, Epidiolex achieved the primary endpoint of a significant reduction in convulsive seizures assessed over the entire treatment period compared with placebo. Despite taking an average of three medicines to reduce seizures, the patients were still having a median of 13 seizures per month.
Despite the surge, shares in the loss-making UK-based group are still down more than 10% over the a year ago. Anecdotal accounts from doctors treating their patients with Epidiolex under that program offer a similar picture to the clinical trial, and also suggest that the drug could eliminate seizures in about one-tenth of patients with Dravet syndrome. Either Epidiolex or the placebo was added to the current anti-epileptic treatment regimen. While the market for Dravet syndrome is quite small, the implication for a pure cannabinoid treatment may be huge, he said.
Following the positive data results, GW Pharmaceuticals stock soared. Another Epidiolex trial for a third type of epilepsy, tuberous sclerosis complex, is scheduled to start soon.
Advertisement
Epidiolex was considered to be well tolerated. Two more pivotal phase 3 trials are ongoing in another rare form of pediatric epilepsy called Lennox-Gastaut Syndrome and are scheduled to readout topline data in second quarter of 2016. The most common side effects included reduced appetite, vomiting, diarrhea, fatigue, fever, drowsiness/sleepiness, upper respiratory tract infections and convulsion.
