With promising Phase III efficacy data, Epidiolex will benefit from the lack of FDA-approved therapies in an orphan indication.
By Maha Elsayed, Analyst
28 May 2016
I am an analyst at Datamonitor Healthcare based at the New York office, where I am part of the CNS and rare disease team...
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GW Pharmaceuticals’ Epidiolex is the latest novel anti-epileptic drug to approach the market. In March 2016, GW Pharmaceuticals announced the results of one of its two pivotal Phase III studies evaluating Epidiolex in the adjunctive treatment of Dravet syndrome (GWEP1332 Part B; ClinicalTrials.gov identifier: NCT02091375). Patients were randomized into two groups, receiving either 20mg/kg/day of Epidiolex or placebo, in addition to their background anticonvulsant therapy. Following a 14-week treatment period, Epidiolex patients had a 39% reduction in monthly seizure frequency when compared to the four-week baseline, contrasting a 13% reduction among the placebo group (p=0.01). Efficacy was established as early as the first month of treatment, and was maintained throughout the treatment period. Following the positive outcome of a Phase III trial evaluating Epidiolex in Dravet syndrome, GW Pharmaceuticals is requesting a pre-New Drug Application (NDA) meeting with the US Food and Drug Administration (FDA) to discuss regulatory submission. Dravet syndrome is a progressive and serious genetic disorder associated with seizures beginning in infancy and continuing throughout life. While stiripentol is approved by the European Medicines Agency for the treatment of Dravet syndrome, there is no FDA-approved treatment in the US. As such, bringing a drug with a new mechanism of action to the market would satisfy a significant unmet need.
Phase III efficacy data for Epidiolex in Dravet syndrome
|Study||Sample size||Target patients||Study design||Dosing tested and duration||Results|
|II/III (NCT02091375; GWEP1332 Part B)||120||Patients aged two to 18 years old with Dravet syndrome, treatment-refractory, on a stable anti-epileptic dug treatment regimen||Randomized, double-blind, placebo-controlled study
Primary endpoint: mean percentage change from baseline convulsive seizure frequency during the treatment period
|Arm 1: Epidiolex 20mg/kg/day
Arm 2: placebo
Duration: 14-week treatment period
|Reduction in monthly seizure frequency Epidiolex: 39%
|Source: GW Pharmaceuticals, 2016; ClinicalTrials.gov; Biomedtracker, 2016|
Source: GW Pharmaceuticals, 2016b
Datamonitor Healthcare’s Epilepsy: Pipeline provides an overview of key pipeline candidates in late-stage development for epilepsy across the US, Japan and five major EU markets (France, Germany, Italy, Spain, and the UK).
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