The Food and Drug Administration (FDA) has approved prescription pharmaceutical cannabidiol (ppCBD) (Epidiolex; GW Pharmaceuticals, Carlsbad, CA) oral solution to treat seizures associated with tuberous sclerosis complex (TSC) in patients age 1 year or more. Along with this new indication, the age range has been expanded to include individuals age 1 and up who experience seizures associated with Lennox-Gastaut syndrome (LGS) or Dravet syndrome (DS).
FDA approval includes a recommended maintenance dose of 25 mg/kg/day for participants, which is supported by data from a phase 3 safety and efficacy study evaluating 25 mg/kg/day of ppCBD. In the trial, treatment with ppCBD reduced seizure frequency by 48% compared with 24% for placebo (P<0.01). Both primary and key secondary endpoints of the trial were met.
PpCBD is the first plant-derived cannabinoid prescription medicine and the only FDA-approved form of cannabidiol (CBD).
“FDA approval of Epidiolex in TSC is a tremendous step forward and our community applauds this positive development,” said Kari Luther Rosbeck, president and chief executive officer of the Tuberous Sclerosis Alliance. “One of the most challenging and frustrating aspects of TSC are seizures that cannot be effectively controlled by existing medications. New treatment options are desperately needed, and this approval adds another option for those impacted by this difficult disease.”
The most common adverse events in those receiving ppCBD in the study (≥10% and greater than placebo) included diarrhea, transaminase elevations, decreased appetite, somnolence, pyrexia, and vomiting. The safety profile observed in this study was generally comparable to that observed in prior studies of ppCBD.
Neha M. Kramer, MD; Jessica Besbris, MD; and Rima M. Dafer, MD, MPH
Ryan Verity, MD; Andrew Kirk, MD, FRCPC; and Gary Hunter, MD, FRCPC, CSCN(EEG)
Zachary S. Gillis, BS, and Michael S. Cartwright, MD, MS