Friday, May 1, 2015

Cannabis-Based Epilepsy Drug (Epidiolex) has Positive News

Epidiolex, is a liquid formulation of highly purified Cannabidiol (CBD) extract which is under trials as a treatment for various orphan pediatric epilepsy syndromes. It has been granted Orphan Drug Designation for the treatment of Dravet syndrome and  Lennox-Gastaut syndrome by US-FDA. 
Epidiolex also known as (GWP42003 oral solution) is as an oily solution containing 100 mg/mL of cannabidiol (CBD) dissolved in excipients, sesame oil, ethanol, sucralose and strawberry flavouring.

GW Pharmaceuticals is developing Epidiolex. 
In a Phase III study, the in-development epilepsy drug slashed rates of seizures by more than 50%, a hopeful sign for the cannabis-derived treatment. In a presentation with American Academy of Neurology, Epidiolex was credited with 54% reduction in number of seizures among patients with severe and/or treatment-resistant epilepsy. 
The study was conducted through an expanded access program, which allows drugmakers to make unapproved products available to patients who have life-threatening diseases with no therapeutic alternatives, and its results by no means stand in for placebo-controlled efficacy data. The study enrolled 213 people from toddlers to adults and dosed them with Epidiolex twice daily. Among the 137 patients who stayed on the drug for 12 weeks, the drug led to a 54% mean reduction in seizures, including a 53% average cut in those with Dravet syndrome and a 55% reduction in sufferers with Lennox-Gastaut syndrome. 
GW Pharma, which is working on Phase III trials studying Epidiolex in Dravet and is planning a late-stage Lennox-Gastaut study in 2015 is very upbeat with these findings. The drug is already enrolled in the FDA's fast-track program, guaranteeing a speedy regulatory review. GW Pharma too plans to conducts a separate late-stage program and investigators believe the open-label study points to potential success for the cannabis-based therapy.

1. Epidiolex and Drug Resistant Epilepsy in Children (CBD). NCT02397863 (retrieved 30-05-2015)
2. A Study of GWP42003 as Adjunctive Therapy in the First Line Treatment of Schizophrenia or Related Psychotic Disorder. NCT02006628 (retrieved 30-05-2015)