Epidiolex, a cannabis-based drug has been used to treat children with Dravet syndrome, a rare form of epilepsy, in GW Pharmaceutical’s (GW) phase three clinical study
In this study, Epidiolex helped provide a significant reduction in convulsive seizures assessed over the entire treatment period compared with placebo, according to GW.
Epidiolex has both Orphan Drug Designation and Fast Track Designation from the US Food and Drug Administration (FDA) in the treatment of Dravet syndrome.
Orrin Devinsky, of New York University Langone Medical Center’s Comprehensive Epilepsy Center, said: “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.
“These data demonstrate that Epidiolex delivers clinically important reductions in seizure frequency together with an acceptable safety and tolerability profile, providing the epilepsy community with the prospect of an appropriately standardized and tested pharmaceutical formulation of cannabidiol being made available by prescription in the future.”
Justin Gover, GW’s chief executive officer, said: “In light of this positive data, we will now request a pre-NDA meeting with the FDA to discuss our proposed regulatory submission
“The positive outcome of this phase three trial is a significant milestone in the development of Epidiolex as a potential new treatment for patients suffering with Dravet syndrome.
“We are excited about the potential for Epidiolex to become the first FDA approved treatment option specifically for Dravet syndrome patients and their families.
“We also look forward with excitement to the upcoming results from the two Phase 3 trials in Lennox-Gastaut syndrome and the second pivotal trial in Dravet syndrome.”
Mary Anne Meskis, executive director of the Dravet Syndrome Foundation, said: “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.”