The United States. The Food and Drug Administration today approved Epidiolex (cannabidiol) [CBD] oral solution for the treatment of seizures associated with component of marijuana rare and severe forms of epilepsy in patients two years of age and older. It is the first drug approved by the FDA to contain a pure drug derived from cannabis. It is also the first FDA approval to treat patients with Dravet syndrome.
CBD is the chemical composition of the Cannabis sativa plant, commonly known as marijuana. However, CBD does not cause the intoxication or euphoria (“high”) that comes with tetrahydrocannabinol (THC).
THC (not CBD) is the main psychoactive component of marijuana.
“This approval recognizes that development programs that properly test the active ingredients in cannabis can lead to important treatments. And the FDA is committed to rigorous scientific research and drug development. We will continue to support and work with rigorous scientific research into the medical use of products derived from cannabis. Product developers are interested in bringing high-quality, safe and effective products to patients, but at the same time, they are ready to take action if we see that CBD products are being illegally marketed with unproven medical claims. Unconfirmed dosages and ingredients and incorrect prescribing can prevent patients from receiving appropriate and recognized treatments to treat serious and life-threatening illnesses.
Dravet syndrome is a rare genetic disorder that occurs in the first year of life and is characterized by febrile seizures. Later, other types of convulsions often occur, including myoclonic convulsions (involuntary muscle contractions).
Lennox-Gastaut syndrome begins in childhood. Seizures come in many forms. People with Lennox-Gastaut syndrome usually start having seizures at a young age, usually between the ages of 3 and 5. More than three-quarters of affected people experience tonic seizures, which cause muscles to contract uncontrollably. Almost all children with Lennox-Gastaut syndrome develop learning difficulties and intellectual disabilities. Many have delayed motor development, such as sitting and crawling. Most people with Lennox-Gastaut syndrome need help with normal activities of daily living.
“The difficult-to-control seizures experienced by patients with Dravet syndrome and Lennox-Gastaut disease have a significant impact on their quality of life,” said Billy Dunn, director of the FDA’s Division of Neurology Products. Drug Testing and Research. “In addition to another important treatment for Lennox-Gastaut patients, this first approval of a drug specifically for Dravet patients will provide an important and necessary advance in the treatment of people with this disease.”
The effectiveness of Epidiolex was studied in three randomized, double-blind, placebo-controlled clinical trials involving 516 patients with Lennox-Gastaut syndrome or Dravet syndrome. Epidiolex taken with other drugs is effective in reducing the frequency of seizures compared to a placebo.
The most common adverse events that occurred in patients treated with Epidiolex in clinical studies were: depression, constipation, and fatigue; elevated liver enzymes; decreased appetite; diarrhea; garbage; fatigue, discomfort, and weakness; insomnia, sleep disorders, and poor quality sleep; and diseases.
Epidiolex should be provided with a patient medication guide that explains important information about the drug’s use and risks. As with all drugs that treat epilepsy, the biggest risks are suicidal thoughts, suicide attempts, feelings of agitation, new or worse depression, seizures, and panic attacks. Epidiolex also tends to cause mild liver damage, but it increases the chance of rare but more serious damage. Severe liver damage may cause nausea, vomiting, abdominal pain, fatigue, loss of appetite, jaundice and/or dark urine.
Under the Controlled Substances Act (CSA), CBD is currently a Schedule I substance because it is a chemical component of the marijuana plant. To support this trend, the company has conducted non-clinical and clinical studies to test the abuse potential of CBD.
The FDA, through the U.S. Department of Health and Human Services, prepares and submits medical and scientific reviews of scheduled substances such as CBD and makes recommendations to the Drug Enforcement Administration (DEA) regarding CSA regulation. An RIA planning decision must be made.
The FDA has given this application a Priority Review designation. Dravet syndrome was called Fast-Track. The description of orphan drugs was given for the indications of Dravet syndrome and Lennox-Gastaut syndrome.
The FDA has granted approval for Epidiolex to GW Research Ltd.
As an agency of the U.S. Department of Health and Human Services, the FDA protects public health by ensuring the safety, efficacy, and safety of human and animal drugs, vaccines, and other biological products and medical devices for human use. The agency is responsible for the safety and security of our nation’s food supply, cosmetics, nutritional supplements, electronic products, and tobacco products.
