NEJM publishes Phase III trial of Epidiolex (cannabidiol) in children with Dravet syndrome. - GW Pharma

GW Pharmaceuticals plc announced that The New England Journal of Medicine has published results from a Phase III study of Epidiolex (cannabidiol) in children with Dravet syndrome. Epidiolex, GW�s lead product candidate and the potential first in a new category of anti-epileptic drugs, is a liquid formulation of purified, plant-derived cannabidiol (CBD), a non-psychoactive cannabinoid, which is being studied for the treatment of a number of rare, severe pediatric-onset epilepsy disorders.

In the study, Epidiolex significantly reduced monthly convulsive seizure frequency compared to placebo in highly treatment-resistant children when added to existing treatment. Treatment with Epidiolex was generally well tolerated, with a safety profile consistent with prior open label experience. There are currently no treatments approved by the FDA for Dravet syndrome, a rare form of epilepsy associated with a high mortality rate and significant developmental delays. Results from this study represent the only well-controlled clinical evaluation of a cannabinoid medication for this devastating and drug-resistant condition. The New Drug Application for Epidiolex remains on track for submission to the FDA in the middle of 2017.

See-"Trial of Cannabidiol for Drug-Resistant Seizures in the Dravet Syndrome."- Orrin Devinsky, M.D., J. Helen Cross, Ph.D., F.R.C.P.C.H., Linda Laux, M.D., Eric Marsh, M.D., Ian Miller, M.D., Rima Nabbout, M.D., Ingrid E. Scheffer, M.B., B.S., Ph.D., Elizabeth A. Thiele, M.D., Ph.D., and Stephen Wright, M.D., for the Cannabidiol in Dravet Syndrome Study Group* N Engl J Med 2017; 376:2011-2020May 25, 2017DOI: 10.1056/NEJMoa1611618.