The U.S. Food and Drug Administration (FDA) on the 25th of June 2018 approved Epidiolex oral solution that is essentially cannabidiol or the active component of marijuana or cannabis. As of now the drug is approved for use in two rare forms of epilepsy - Lennox-Gastaut syndrome and Dravet syndrome. The approval comes for individuals over two years of age.
Cannabis based drugs contain cannabidiol or CBD that comes from the Cannabis sativa plant or marijuana. The unique property of CBD is that it does not cause euphoria that is associated with cannabis. It also does not lead to intoxication that comes from another component of cannabis - tetrahydrocannabinol (THC). Epidiolex is the first FDA approved drug that contains a cannabis component and this is also the first approved drug for Dravet syndrome.
Lennox-Gastaut syndrome is a rare form of epilepsy that shows different types of seizures. It usually starts by the age of 3 to 5 years. The seizures are usually tonic in type and there is severe intellectual developmental delays and retardation of motor skills. Dravet syndrome is a genetic form of epilepsy that usually manifests during the first year of life. The child usually shows frequent febrile seizures that later on lead to other forms of seizures such as myoclonic seizures. There may be several episodes of status epilepticus which may be life threatening emergency situations. These frequent seizures affect the growth and intellectual development of the child.
According to a statement made by FDA Commissioner Scott Gottlieb, “This approval serves as a reminder that advancing sound development programs that properly evaluate active ingredients contained in marijuana can lead to important medical therapies. And, the FDA is committed to this kind of careful scientific research and drug development.” “Controlled clinical trials testing the safety and efficacy of a drug, along with careful review through the FDA’s drug approval process, is the most appropriate way to bring marijuana-derived treatments to patients. Because of the adequate and well-controlled clinical studies that supported this approval, prescribers can have confidence in the drug’s uniform strength and consistent delivery that support appropriate dosing needed for treating patients with these complex and serious epilepsy syndromes. We’ll continue to support rigorous scientific research on the potential medical uses of marijuana-derived products and work with product developers who are interested in bringing patients safe and effective, high quality products. But, at the same time, we are prepared to take action when we see the illegal marketing of CBD-containing products with serious, unproven medical claims. Marketing unapproved products, with uncertain dosages and formulations can keep patients from accessing appropriate, recognized therapies to treat serious and even fatal diseases,” he said.
Billy Dunn, M.D., director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research in his statement said that because there is severe difficulty in controlling seizures in these conditions, there is a wide impact on the quality of lives of these children. “In addition to another important treatment option for Lennox-Gastaut patients, this first-ever approval of a drug specifically for Dravet patients will provide a significant and needed improvement in the therapeutic approach to caring for people with this condition,” he said.
This approval came after the manufacturers of Epidiolex provided data from three randomized, double-blind, placebo-controlled clinical trials. A total of 516 patients with either Lennox-Gastaut syndrome or Dravet syndrome were included. Patients were randomized to receive either Epidiolex or placebo along with other standard therapy. Seizure frequency was found to be reduced with Epidiolex compared to placebo.
Common side effects with the use of these drugs include sedation, lethargy, rash, diarrhea, fatigue, reduced appetite, sleep disorders, poor sleep quality and raised liver enzymes. More serious side effects to look out for include suicidal thoughts, panic attacks, depression etc. The approval states that the drug needs to be dispensed along with a Patient Medication Guide that would provide necessary safety information regarding the drug. At present CBD is a Schedule I substance under the Controlled Substances Act (CSA) because of its origin from the cannabis plant. Abuse potential of the drug was also studied in details by the manufacturers.
Epidiolex received Priority Review designation for its application for approval and Fast Track designation for its use in Dravet’s syndrome. It received an Orphan drug status for both the indications. Approval for Epidiolex was granted to British drug makers GW Research Ltd. The Drug Enforcement Administration (DEA) has been given 90 days to schedule the medication.
According to Stephen Schultz, vice president of investor relations for GW Pharmaceuticals the drug would be launched this fall tentatively after the DEA schedules it. The drug, he said would be produced in the UK and exported to USA. Pricing would also be set after the DEA schedules the drug.