- Pharmaceutical formulation of cannabidiol significantly reduced convulsive seizure frequency in children on multiple anti-epileptic drugs with poor seizure control -
- First well-controlled clinical study of cannabidiol in Dravet syndrome, a rare, severe type of epilepsy with no
There are currently no treatments approved by the
"Dravet syndrome is one of the most difficult types of epilepsy to treat and many of the children in this study were experiencing dozens, even hundreds, of seizures per month despite taking multiple concurrent anti-epileptic medications," said
"The publication of these highly-anticipated positive results represents an important milestone for the Dravet syndrome community in that it provides hope that a new treatment option is within sight for this rare and devastating disease," said
The study randomized 120 children ages two to 18 years (mean age 9.8), with Dravet syndrome whose seizures were not controlled by
their current anti-epileptic regimen, to receive either Epidiolex (20mg/kg/day) or placebo in addition to standard treatment. Conducted in 23 study centers in
Over the 14-week treatment period, patients taking Epidiolex had a significantly greater median reduction in convulsive seizures (39 percent) compared to placebo (13 percent). The estimated median treatment difference between groups was 23 percent (p=0.01). The proportion of patients who had a 50 percent or better reduction in convulsive seizure frequency was 43 percent with Epidiolex versus 27 percent with placebo (p=0.08). The study also measured improvement on the Caregiver Global Impression of Change (CGIC) scale. Sixty-two percent of patients treated with Epidiolex were rated as improved in overall condition on the CGIC compared to 34 percent of the placebo group (p=0.02). Additionally, more patients became seizure-free on Epidiolex than placebo (5 percent vs 0 percent: p=0.08) and total monthly seizure count was significantly reduced with Epidiolex (p=0.03).
Epidiolex was generally well tolerated in the trial. The most common adverse events (AEs) ( > 10 percent) were somnolence, diarrhea, decreased appetite, fatigue, vomiting, pyrexia, lethargy, convulsion, upper respiratory tract infection. Of the 93 percent of patients on Epidiolex that experienced an AE, 84 percent reported it to be mild or moderate. Ten patients on Epidiolex experienced a serious adverse event compared with three patients on placebo. Eight patients on Epidiolex discontinued treatment due to adverse events compared with one patient on placebo. Elevations in liver enzymes occurred in 12 patients taking Epidiolex and one patient taking placebo, all of whom were on concomitant valproic acid. Four of these patients withdrew from the trial, three on Epidiolex and one on placebo. In the remaining nine patients taking Epidiolex, elevations returned to normal while on treatment.
"The publication of results from this landmark study by
About Dravet Syndrome
Dravet syndrome is a severe infantile-onset and highly treatment-resistant epileptic syndrome frequently associated with a genetic mutation in sodium channels. Onset of Dravet syndrome occurs during the first year of life in previously healthy and developmentally normal infants. Initial seizures are often temperature related, severe, and long-lasting. Over time, people with Dravet syndrome can develop multiple types of seizures, including tonic-clonic, myoclonic, and atypical absences and are prone to bouts of prolonged seizures called status epilepticus, which can be life threatening. Risk of premature death including SUDEP (sudden unexpected death in epilepsy) is elevated in people with Dravet syndrome. Additionally, the majority will develop moderate to severe intellectual and development disabilities and require lifelong supervision and care. There are currently no FDA-approved treatments and nearly all patients continue to have uncontrolled seizures and other medical needs throughout their lifetime.
Epidiolex, GW's lead cannabinoid product candidate, is a liquid formulation of plant-derived cannabidiol (CBD), which is in development for the treatment of a number of rare childhood-onset epilepsy disorders. GW has conducted extensive pre-clinical research of CBD in epilepsy since 2007. This research has shown
that CBD has significant anti-epileptiform and anticonvulsant activity using a variety of in-vitro and in-vivo models and efficacy in reducing seizures in acute animal models of epilepsy. To date, GW has received Orphan Drug Designation from the
Founded in 1998, GW is a biopharmaceutical company focused on discovering, developing and commercializing novel therapeutics from its proprietary cannabinoid product platform in a broad range of disease areas. GW is advancing an orphan drug program in the field of childhood-onset epilepsy with a focus on Epidiolex® (cannabidiol), which is in Phase 3 clinical development for the treatment of Dravet syndrome, Lennox-Gastaut syndrome,
This news release contains forward-looking statements that reflect GW's current
expectations regarding future events, including statements regarding financial performance, the timing of clinical trials, the timing and outcomes of regulatory or intellectual property decisions, the relevance of GW products commercially available and in development, the clinical benefits of Epidiolex® and the safety profile and commercial potential of Epidiolex. Forward-looking statements involve risks and uncertainties. Actual events could differ materially from those projected herein and depend on a number of factors, including (inter alia), the success of GW's research strategies, the applicability of the discoveries made therein, the successful and timely completion of uncertainties related to the regulatory process, and the acceptance of Epidiolex and other products by consumer and medical professionals. A further list and description of risks and uncertainties associated
with an investment in GW can be found in GW's filings with the
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1 Devinsky O, Cross JH, Laux L, et al. Trial of cannabidiol for drug-resistant seizures in the Dravet syndrome. N Engl J Med 2017; 376;2011-20.
2 Berkovic, SF. Editorial: Cannabinoids for epilepsy - real data, at last. N Engl J Med 2017; 376;2075-76.
GW Pharmaceuticals plc Stephen Schultz, VP Investor Relations 401 500 6570 U.S.Media Enquiries: Sam Brown Inc. Healthcare Communications Christy Curran615 414 8668 Mike Beyer312 961 2502
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