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