Pharmaceutical-grade cannabidiol (Epidiolex) as adjunctive treatment of seizures associated with Lennox-Gastaut syndrome (LGS) or Dravet syndrome received positive words from FDA staff in briefing documents issued in advance of an FDA advisory committee meeting that will be held Thursday.
The risk-benefit profile established by three clinical trials in LGS and Dravet syndrome “appears to support approval,” they wrote. The trials demonstrated clinically meaningful and statistically significant reductions in seizure frequency and their results “provide substantial evidence” of cannabidiol’s effectiveness in treating LGS and Dravet syndrome seizures, according to the review.
In light of this, possible risks associated with cannabidiol appeared acceptable: “Although the risk of liver injury has the potential to be serious, the observed risk can be appropriately managed with inclusion of relevant language in labeling, education of prescribers regarding the risk of transaminase elevation and need for monitoring of liver enzyme levels, and further characterization of the risk in the post-market setting,” the document said.
The FDA is expected to decide on GW Pharmaceuticals’ application by June 27. The product would be the first cannabis-derived drug approved in the U.S.
LGS and Dravet syndrome are rare, severe, refractory epilepsy syndromes that emerge early in childhood. Both are linked to higher rates of mortality, primarily due to status epilepticus and sudden unexpected death in epilepsy patients (SUDEP). Cannabidiol, a cannabinoid prepared from marijuana, is structurally unrelated to other anti-seizure medications and currently is a Schedule I drug. Its anticonvulsant mechanism is unknown but does not appear to involve cannabinoid receptors.
Thursday’s session of the Peripheral and Central Nervous System Drugs Advisory Committee will vote on only one question: whether the benefit-risk profile of the cannabis derivative is favorable for the treatment of seizures associated with LGS and Dravet syndrome in people 2 years old and older.
The application and