GW Pharma’s Dravet Syndrome Drug (Epidiolex) in Phase III with Positive Results
London based Pharmaceutical company GW Pharma had announced positive results in regard to its drug Epidiolex (cannabinoid) for the treatment of Dravet Syndrome. The company has been long focused on treating the Dravet Syndrome, a rare and special kind of epilepsy in children for which there is no U.S. FDA approved treatment available so far.
In this course of study, Epidiolex achieved the expected decrease in seizure rate as compared to the traditional placebo treatment used in the treatment of this syndrome. Epidiolex has both Orphan Drug designation and Fast Track designation by the U.S. FDA.
The Phase III study worked with 120 randomly selected patients. The patients were studied in two groups, one administered Epidiolex 20 mg per kg per day (61 patients) and the other under simple placebo treatment (59 patients). Epidiolex or the placebo was added to the ongoing antiepileptic drug (AED) course. On an average, patients were taking 3 AEDs having already failed 4 other AEDs. In this study, patients taking Epidiolex achieved a median reduction in monthly convulsive seizures of 39 percent compared with a reduction on placebo of 13 percent, which was highly statistically significant (p=0.01). A series of sensitivity analyses of the primary endpoint confirmed the strength of this result. The difference between Epidiolex and placebo emerged during the first month of treatment and was sustained during the entire treatment period.
The most common side effects suffered by patients drug include: somnolence, diarrhea, decreased appetite, fatigue, pyrexia, vomiting, lethargy, upper respiratory tract infection and convulsion. Of those patients on Epidiolex that reported an adverse event, 84 percent reported it to be mild or moderate. Ten patients experienced a serious adverse event compared with three patients on placebo. Eight patients on Epidiolex discontinued treatment due to adverse events compared with one patient on placebo.
“The results of this Epidiolex pivotal trial are important and exciting as they represent the first placebo-controlled evidence to support the safety and efficacy of pharmaceutical cannabidiol in children with Dravet syndrome, one of the most severe and difficult-to-treat types of epilepsy,” said Orrin Devinsky, M.D., of New York University Langone Medical Center’s Comprehensive Epilepsy Center.
Given to the success of this first Phase III trial, GW is conducting a second Phase III trial in Dravet syndrome which is recruiting 150 patients. Apart from Dravet syndrome trials, GW is conducting global pivotal clinical trial program to decipher the efficacy of the drug for another rare form of epilepsy-Lennox-Gastaut syndrome whose results are expected in second quarter of 2016. A Phase III trial for Tuberous Sclerosis complex-a pediatric epilepsy will also start in the other half of 2016. The current ongoing developments on Epidiolex are showing a bright future for the treatment of various forms of epilepsy.
This visualization of the endocannabinoid signaling system will help you in understanding the molecular mechanism of Epidiolex (Copyright: Leanne Chan 2011).
Featured image credit: Dravet Syndrome Diagnosis. Medical Concept. © tashatuvango (Stock Photo ID: 93119975)