Advisory Committee Supports Approval of Oral Cannabidiol for Dravet Syndrome and Lennox-Gastaut Syndrome
The Peripheral and Central Nervous System Drugs Advisory Committee of the Food and Drug Administration (FDA) unanimously recommended supporting the approval of the investigational cannabidiol oral solution (CBD)(Epidiolex; Greenwich Biosciences, Carlsbad, CA) for the adjunctive treatment of seizures associated with Lennox-Gastaut syndrome (LGS) and Dravet syndrome in patients two years of age and older.
If approved, CBD will be groundbreaking in multiple ways. It will be the first If approved pharmaceutical formulation a compound derived from cannabis (marijuana) that lacks the psychotropic effects (high) associated with marijuana. It will also be the first in a new category of both antiepileptic and neurologic drugs, targeting the cannabinoid receptor.
More than 90% of patients with LGS or Dravet syndrome have multiple seizures per day, which puts them at constant risk for falls and injury. The sheer volume of seizures per day makes treatment with existing antiepileptic drugs suboptimal at best. If approved, CBD will also be the first-ever FDA-approved medicine for Dravet syndrome patients.
The advisory board heard results of a robust clinical development program that included 3 phase 3 clinical trials and an open label extension study. In phase 3 studies, CBD as an adjunct to other antiepileptic therapies significantly reduced seizure frequency for patients with LGS and Dravet syndrome. Most adverse events reported were mild or moderate.
“As a physician who treats LGS and Dravet syndrome, I know that patients and their families usually face significant difficulties getting seizures under control using existing therapies,” said Elizabeth Thiele, MD, PhD, director of pediatric epilepsy at Massachusetts General Hospital, professor of Neurology at Harvard Medical School and a primary investigator for one of GW’s and Greenwich’s studies in LGS patients. “The results from these studies suggest that this pharmaceutical formulation of cannabidiol may provide hope for a new treatment option that may be effective for some patients.”