The U.S. Food and Drug Administration (FDA) has approved Epidiolex to treat Dravet and Lennox-Gastaut syndromes, making history as the first approved therapy for Dravet, as well as the first marijuana-derived treatment to receive FDA approval for any indication. It is used to treat two rare forms of epilepsy in patients 2 years of age and older.
The FDA's action also may bring hope to patients with other rare diseases marked by seizures, like Angelman syndrome.
It is estimated there are up to 18,500 patients with LGS, and 20,000 of Dravet Syndrome in the United states.
Earlier, the agency has approved the synthetic versions of certain CBD chemicals which were found in marijuana plant for other use. Because of this, parents don't have to worry about their children actually feeling the effects of THC from the oral solution medication.
The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices.
Rite Aid told CNNMoney it would sell Epidiolex once the DEA schedules it.
During clinical trials, the researchers were able to observe the following effects: sleepiness, sedation, lethargy, fatigue, elevated liver enzymes, decreased appetite, diarrhea, rash, malaise, weakness, insomnia, poor quality sleep, and various infections.
It's unclear at this time whether Epidiolex receiving FDA approval will move marijuana to a different class of drugs in the future, or how this will affect CBD research in the future.
FDA Commissioner Scott Gottlieb further added: "This is the approval of one specific CBD medication for a specific use". FDA has determined that CBD has negligible abuse potential. For instance, a clinical trial is underway to test whether CBD can be an effective treatment for people with post-traumatic stress disorder and alcohol use disorder.
As part of the approval process, EPIDIOLEX must be rescheduled from its current Schedule I before it can be made available to patients.
As a result, Epidiolex's launch remains at the discretion of the DEA, which must now evaluate the drug and consider reclassifying it as a substance that has medical properties, so as to allow GW to begin selling it. Marketing unapproved products, with uncertain dosages and formulations, can keep patients from accessing appropriate, recognized therapies to treat serious and even fatal diseases.