A UK-headquartered biopharmaceutical company has announced positive results from the first Phase 3 study of its cannabidiol (CBD) based drug for the treatment of a severe form of epilepsy.
Dravet syndrome, also known as severe myoclonic epilepsy of infancy (SMEI), usually appears at around six months of age; with seizures worsening over time. The condition can be accompanied by hyperactivity, impulsiveness, autistic type behaviours and other developmental disorders.
Dravet syndrome is a rare genetic disorder that affects an estimated 1 in every 20,000-40,000 births, and patients often require a great deal of ongoing care.
GW Pharmaceuticals says its investigational medicine Epidiolex achieved a significant reduction in convulsive seizures in Dravet’s sufferers assessed over the entire treatment period compared with placebo.
The Phase 3 study randomized 120 patients into two groups of trial participants with an average age of 10 years and a median baseline convulsive seizure frequency of 13 events per month.
One group received Epidiolex at a dosage of 20mg/kg/day and the other, a placebo. Patients in both groups continued with any current anti-epileptic drug treatment regimens.
The group given Epidiolex achieved a median reduction in monthly convulsive seizures of 39 percent; compared with a reduction 13 percent for the placebo group.
“We are excited about the potential for Epidiolex to become the first FDA approved treatment option specifically for Dravet syndrome patients and their families,” stated Justin Gover, GW’s Chief Executive Officer.
The company is now seeking a pre-NDA meeting with the U.S. Food and Drug Administration to discuss its proposed regulatory submission.
The Dravet Syndrome Foundation was understandably excited with the news of the study results.
“Dravet syndrome is one of the most catastrophic types of epilepsy in children and safe and effective treatments are desperately needed. We are thrilled to learn of these positive results, which bring much needed hope to the children and families who have been living with these debilitating seizures,” said Mary Anne Meskis, the Foundation’s Executive Director.
Further information regarding the study can be viewed here.
In December last year, another study presented evidence that the cannabidiol-based medicine can be effective in treating epilepsy in children.
Cannabidiol is a non-intoxicating compound extracted from medical cannabis that is claimed to have a number of other beneficial attributes; including antibacterial properties, blood sugar level reduction, pain and anxiety relief, and anti-inflammatory properties to name a few.
Cannabidiol can also be extracted from industrial hemp.