FDA Panel Recommends GW Pharma’s Cannabis-based Drug for Seizure Disorders

The U.S. Food and Drug Administration (FDA)’s Peripheral and Central Nervous System Drugs Advisory Committee unanimously recommended the approval of London, UK-based GW Pharmaceuticals’ Epidiolex for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS) and Dravet syndrome.

Epidiolex is a cannabidiol oral solution (CBD). If approved, it would be the first pharmaceutical formulation of plant-based CBD, a cannabinoid associated with marijuana. It would also be the first in a new category of anti-epileptic drugs (AEDs). It does not contain THC, the chemical in marijuana that causes the "high."

LGS and Draven syndrome develop in childhood. They are both forms of epilepsy with high morbidity and mortality rates. More than 90 percent of patients with these diseases have multiple seizures daily. Epidiolex, if approved, would be the first FDA-approved drug for Dravet syndrome.

"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," said Justin Gover, GW’s chief executive officer, in a statement. "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."

The recommendation came out of Phase III trials that added Epidiolex to other existing antiepileptic therapies. The drug cut the frequency in seizures in LGS and Dravet syndrome patients.

FDA Advisory Committees are independent, and the agency does not have to follow the recommendation, although it usually does. The PDUFA date for the final decision on Epidiolex is June 27, 2018.

The panel was made up of 13 independent researchers. They felt that the benefit-risk profile of the drug was favorable.

Some analysts have projected Epidiolex’s peak annual sales between $1 and $3 billion. In GW’s last fiscal year, it reported $11 billion in revenue, mostly from sales of THC-CBD drug Sativex for multiple sclerosis. It is approved for sale in about a dozen countries outside the U.S. Although GW hasn’t discussed pricing information, analysts have estimated the likely costs would be $30,000 to $60,000 per year if insurance doesn’t cover it.

Although there have been questions as to whether GW has the commercial infrastructure to launch the drug on its own, Gover denies it. Referring to $300 million the company recently raised from a secondary public offering, he told The Cannabist, "We have the resources we need. Nobody knows cannabinoid science better than we do. We’re very much going at it alone and very happy to do so."

Evaluate Group’s EP Vantage, a biotech research firm, noted, "The group has been busy building its commercial team and will be targeting 4,000-5,000 doctors in the U.S., a big undertaking for a small company."

This news follows recent reports of increased activity in the U.S. for medical marijuana companies, specifically Oak Park, Illinois-based PharmaCannis, a PharmaCann company. PharmaCannis takes a biopharmaceutical approach to medical marijuana, developing capsules, instead of smoking marijuana. This provides more precise dosing, which is difficult to do with smoking.

Industry observers speculate that if the FDA approves Epidiolex in the U.S., it could lead to broader research-and-development efforts of cannabis-based therapeutics in the country.

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