On April 19th, 2018 the Peripheral and Central Nervous System Drugs Advisory Committee (PCNS) convened to review new drug.
On April 19th, 2018 the Peripheral and Central Nervous System Drugs Advisory Committee (PCNS) convened to review new drug application (NDA) 210365 for GW Pharmaceuticals’ novel seizure drug, Epidiolex (cannabidiol). Cannabidiol (CBD) is an oral solution indicated for seizures associated with Lennox-Gastuat syndrome (LGS) and Dravet syndrome (DS) in patients at least 2 years old. These conditions can cause a person to have multiple seizures a day and puts them at risk for falling or further injury at any time.
The mechanism behind the anticonvulsive effect produced by Cannabidiol is unknown, but the drug does not seem to interact with cannabinoid receptors. The sponsor presented data from 3 randomized, double blind, placebo-controlled trials. These 3 multicentered trials spanned 14 weeks; 2 of the trials were conducted with Lennox-Gastaut syndrome patients and the other with Dravet syndrome patients. Data presented on efficacy was clinically meaningful and statistically significant. Additional studies were conducted for safety.
The most commonly observed adverse events occurred predominantly in the CBD group and included somnolence, sedation, decreased appetite, diarrhea, transaminase elevations and infections like pneumonia. Most adverse events were considered mild to moderate; severe cases due to transaminase elevation, somnolence, lethargy and infections were reported. Severe transaminase elevations never lead to liver failure or death, despite severity. Only two more patients in the cannabidiol group compared to the control group experienced serious adverse events.
The panel members unanimously voted in support of cannabidiol’s use in its intended patient population (13 Yes and 0 No). Panel members expressed that this new treatment option could provide hope for people affected by the debilitating diseases. One panelist stated that the risk associated with the treatment is acceptable considering the severity of the life-threatening conditions it is meant to treat. Overall, the committee indicated that the risks could be adequately mitigated with proper labeling and education.
The agency is not obligated to follow its panel’s recommendation, but normally does so. On June 25th, 2018, the FDA approved Epidiolex oral solution for LGS and DS in patients 2 years of age and older. This is the first FDA approved medication for the treatment of DS and one of the few approved for LGS. The sponsor is looking to do further research into the use of this medication in other populations.
ISS has almost two decades of experience in developing regulatory strategies including support for FDA Advisory Committee meetings. We are involved in more FDA AdComms per year than even the largest pharmaceutical companies. For more information on how ISS can help you prepare for your next meeting, contact email@example.com
This post was authored by Sami Mourad. Sami is a summer intern at ISS and a 3rd year pharmacy student at the Rutgers University Ernest Mario School of Pharmacy.
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