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16:31 PM

FDA Panel Approves Cannabidiol-Based Epilepsy Drug Studied at Cook Children’s

An epilepsy treatment derived from the cannabis plant took a major step toward approval from the Food and Drug Administration today. Epidiolex, an oil-based purified cannabidiol (CBD), has been studied specifically in two rare epilepsy syndromes - Lennox Gastaut and Dravet Syndrome.

A panel convened by the FDA voted 13-0 that the agency should approve the drug as treatment of these two rare forms of epilepsy. The FDA will make a final decision to approve the medicine by June 27.

Cook Children’s participated in several of GW Pharmaceutical’s clinical trials of Epidiolex used to treat children over the age of 2 with Lennox Gastaut and Dravet and continues to enroll children in a trial of the drug for treatment of seizures in Tuberous Sclerosis Syndrome. “The trials were the largest of their kind in the world and have provided the gold-standard evidence needed to prove cannabidiol can be effective as a treatment for epilepsy,” said M. Scott Perry MD, Medical Director of Neurology and principle investigator for the trials performed at Cook Children’s.

The FDA gave the drug a favorable review, stating that it provides “substantial evidence” of the drug’s effectiveness in treating Lennox-Gastaut syndrome and Dravet syndrome.

“Although the review is still ongoing, the risk-benefit profile established by the data in the application appears to support approval of cannabidiol for the treatment of seizures associated with LGS [Lennox-Gastaut syndrome] and DS [Dravet syndrome]," the FDA said in a report.

Dr. Perry explains that Epidiolex is an essentially pure CBD substance produced under strict standards to ensure the drug is the same with every batch. Once approved, the drug would be obtained from a pharmacy, prescribed by a doctor and more likely to be covered by insurance.

“My hope is this will be the first of many drugs derived from the cannabis plant,” Dr. Perry said. “This drug has gone through a rigorous process to be approved and I think it will help people feel safe taking the medication. I hope it also encourages more research into what the hundreds of other substances in the cannabis plant can do for patients.”

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Get to know M. Scott Perry, M.D.

I joined the Neurosciences Program of Cook Children's in 2009 as a pediatric epileptologist, then served as the Medical Director of the Epilepsy Monitoring Unit and Tuberous Sclerosis Complex clinic before assuming the role of Medical Director of Neurology in 2016. My clinical and research interests focus on the treatment of childhood onset epilepsy, specifically those patients with uncontrolled epilepsy or those for which the cause has not been determined. I have an intense interest in the use of surgical therapies to treat and cure epilepsy. The majority of my research has investigated the use of multimodal imaging techniques to localize seizure onset, as well as the description of patient and disease characteristics that predict favorable outcomes from surgical therapies. The pool of candidates which may benefit from surgical therapy continues to expand and I came to Cook Children's specifically because the staff of the Epilepsy Monitoring Unit and Comprehensive Epilepsy Program were dedicated to improving the care of children with epilepsy through cutting-edge techniques, research, and concern for their patients' well-being. Click to learn more.