As expected, an FDA advisory panel has backed approval of GW Pharmaceuticals/Greenwich Bioscience’s Epidiolex for treating seizures associated with Lennox-Gastaut syndrome and Dravet syndrome.
Signalling a major leap towards market approval, the drug won unanimous support from the Peripheral and Central Nervous System Drugs Advisory Committee for use in patients aged two years of age and older.
If approved, Epidiolex (cannabidiol) would be the first pharmaceutical formulation of purified, plant-based CBD, a cannabinoid lacking the high associated with marijuana, and the first in a new category of anti-epileptic drugs (AEDs), GW noted.
LGS and Dravet syndrome, are devastating forms of childhood-onset epilepsy with high morbidity and mortality rates and a significant burden on families and caregivers. More than 90 percent of patients with LGS or Dravet syndrome have multiple seizures per day, which puts them at constant risk for falls and injury.
Clinical trials demonstrated significant reductions in the monthly seizure rate of patients with either condition taking Epidiolex, while the drug was found to be generally well tolerated with most adverse events reported as mild or moderate.
“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,” commented Elizabeth Thiele, director of paediatric 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 Committee’s decision is not binding, but the FDA generally tends to follow its advice. A final decision is expected by June 27.