A new prescription drug manufactured by UK-based GW Pharmaceuticals PLC might be the first cannabis-based prescription medicine to be approved by the United States Food and Drug Administration.
An FDA advisory panel recommended the agency’s approval of the cannabis-derived Epidiolex for the treatment of rare forms of epilepsy. The committee voted 13-0 in support of Epidiolex after deciding that the drug’s risk-benefit profile was favorable.
Epidiolex is a purified formulation of cannabidiol or CBD, which is used in the treatment of Dravet syndrome and Lennox-Gastaut syndrome in patients aged two years old and above. These two rare medical conditions are characterized by frequent seizures that are difficult to treat.
The panel, called the Peripheral and Central Nervous System Drugs Advisory Committee, did not express any concerns with regard to the effectiveness or the safety of the medicine. It also congratulated the FDA, GW Pharmaceuticals, and the patients and their families for being able to present solid evidence in support of the drug.
The FDA is expected to make its decision on the drug by the end of June. And because the FDA is known to usually follow the advice of its panel, many are optimistic that the agency will approve Epidiolex for sale and distribution in the U.S.
According to Dr. Billy Dunn, director of FDA-Division of Neurology Products, the agency is now reviewing the drug on an expedited timeline.
Also reviewing Epidiolex is the European Medicines Agency, which accepted GW Pharmaceuticals’ application earlier this year.
GW Pharmaceuticals says that the cannabis-derived formulation is made from a proprietary strain of the cannabis plant that is designed to minimize the components that create high while maximizing its therapeutic component. The drug is taken orally.
There have been clinical studies that found that CBD reduced the frequency of seizures in patients. One study involving 120 children and young patients suffering from Dravet syndrome found that CBD significantly reduced the median frequency of the convulsive seizures from 12.4 a month prior to treatment, to 5.9 a month after.
Some of the parents whose children took part in the clinical studies even attested before the panel that the drug helped reduce their kids’ seizures and therefore improved their quality of life.
There are adverse effects associated with Epidiolex, including abnormal liver function test results and gastrointestinal problems. However, the panel pointed out that the risks were manageable as long as the prescribing label in the drug includes cautionary language and as long as the FDA monitors the risk of liver risk after Epidiolex has started selling in the market.
Additionally, FDA officials said that Epidiolex does not have a potential for abuse, citing minimal euphoric effects reported by patients who were involved in the clinical studies and who have taken the drug.
Harriet de Wit, FDA committee member and professor at University of Chicago’s psychiatry and behavioral neuroscience department, said that she believes the case has already been made and that she thinks there is a real need for such drug. She also added that she thinks the safety concerns are negligible.
The Drug Enforcement Administration (DEA) currently considers Epidiolex as a Schedule I drug, which means it is illegal and strictly prohibited. However, if the FDA gives the drug its stamp of approval, the agency will also be making a recommendation that the DEA reschedule the drug.
If Epidiolex gets rescheduled, GW Pharmaceuticals will be able to make it available to doctors as soon as possible. Hopefully, it will be in the second half of 2018. The pharmaceutical company is proposing that patients start with an initial target daily dose of 10 mg/kg, with adjustments of up to 20 mg/kg based on their clinical response and tolerability.