FDA Committee Recommends Approval of Novel Canniboidal for Epilepsy

An FDA committee last week unanimously recommended the approval of a New Drug Application (NDA) for a novel cannabidiol (CBD) oral solution for 2 rare forms of epilepsy.

Epidiolex is being evaluated as an adjunctive treatment for seizures associated with Lennox-Gastaut syndrome (LGS) and Dravet syndrome in patients aged 2 years and older. Epidiolex would be the first pharmaceutical formulation of purified, plant-based CBD to gain FDA approval and the first treatment approved for Dravet syndrome.

Epidiolex, which is the first in a new category of anti-epileptic drugs, does not carry the high associated with marijuana, according to a manufacturer press release.

“We are pleased by the Advisory Committee’s unanimous recommendation to approve Epidiolex, which would provide an important treatment option for patients with LGS and Dravet syndrome, two of the most severe and treatment-resistant forms of epilepsy,” Justin Gover, GW Pharmaceuticals chief executive officer, said in a press release. “This favorable outcome marks an important milestone in our company’s unwavering commitment to address the significant unmet need for patients with LGS and Dravet syndrome and our resolve to study Epidiolex under the highest research and manufacturing standards. We look forward to our ongoing discussions with the FDA as it continues to review the Epidiolex NDA."

LGS and Dravet syndrome, which typically manifest in childhood, are devastating forms of epilepsy that carry high morbidity and mortality rates. In excess of 90% of patients with LGS or Dravet syndrome experience multiple seizures per day, according to the release.

It remains a challenge for patients with these conditions to reduce the volume of dangerous seizures with currently available treatments, according to the release.

In phase 3 trials, Epidiolex in combination with other antiepileptic therapies was found to significantly reduce the frequency of seizures in patients with LGS and Dravet syndrome, according to the release. Epidiolex was shown to be generally well tolerated, with most adverse events reported as mild or moderate, according to the release.

Thus far, Epidiolex has been granted Orphan Drug Designation for the treatment of Dravet syndrome, LGS, tuberous sclerosis complex, and infantile spasms, as well as Fast Track Designation from the FDA for Dravet syndrome and a conditional grant of rare pediatric disease designation by the FDA.

“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,” Elizabeth Thiele, MD, PhD, director of pediatric epilepsy at Massachusetts General Hospital, professor of Neurology at Harvard Medical School, said in the press release. “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.”

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