British-based GW Pharmaceuticals received more good news regarding its cannabis-based drug Epidiolex.
The drug maker announced positive Phase 3 data from a randomized study testing the treatment, which is a liquid formulation of purified, plant-derived cannabidiol (CBD). Researchers randomized120 treatment-resistant children ages two to 18 years diagnosed with a rare, severe form of epilepsy called Dravet syndrome.
Investigators gave patients either 20 mg per kilogram of body weight per day of GW’s treatment or placebo in addition to standard therapies for this condition over a 14-week period. Study participants had previously tried a median of four anti-epileptic drugs and were taking a median of three of them during the study.
Results indicated that Epidiolex significantly reduced the frequency of monthly convulsive seizure frequency better than placebo in those children receiving the drug along with standard treatment.
The experimental treatment caused a 39 percent median reduction in convulsive seizures versus 13 percent in the placebo group.
Overall, the drug was able to improve patients’ conditions and helped more individuals become seizure-free than placebo.
“Dravet syndrome is one of the most difficult types of epilepsy to treat and many of the children in this study were experiencing dozens, even hundreds, of seizures per month despite taking multiple concurrent anti-epileptic medications,” said lead study author Dr. Orrin Devinsky, M.D., of NYU Langone Medical Center’s Comprehensive Epilepsy Center. These results suggest that Epidiolex can provide clinically meaningful benefits and I look forward to the prospect of an appropriately standardized and tested pharmaceutical formulation of cannabidiol available as a treatment option for these patients.”
These study results, published in The New England Journal of Medicine, are the only well-controlled clinical evaluation of a cannabinoid medication for this type of condition.
Epidiolex has previously received a fast track designation from the U.S. Food and Drug Administration (FDA) for Dravet Syndrome and an Orphan Designation from the European Medicines Agency for Dravet and another condition called Lennox-Gastaut Syndrome.
The company is still on track to submit a new drug application to the FDA by the middle of this year.
Filed Under: Drug Discovery