The United States Food and Drug Administration (FDA) has officially approved the use of Epidiolex, a cannabis-based drug designed to treat two severe forms of epilepsy. This makes Epidiolex the first cannabis-derived medicine to be legalized by the U.S. federal government.
Epidiolex, which is an oral prescription medication, was developed by UK-based GW Pharmaceuticals PLC. It can be used to treat Dravet syndrome and Lennox-Gastaut syndrome in patients who are over two years old. These two are rare medical conditions that are characterized by frequent yet hard-to-treat seizures.
Dravet syndrome is a rare genetic brain dysfunction that starts in the first year of life. It is associated with fever-related seizures, hyperactivity, poor language development and motor skills, and difficulty relating to others. Dravet syndrome has a high mortality rate, with young patients dying before they reach 10 years old.
Lennox-Gastaut syndrome, on the other hand, is a form of epilepsy characterized by tonic seizures, wherein muscles uncontrollably contract. It is also an early-onset disease, usually starting between the ages of three and five, and is linked with intellectual disabilities, learning problems, and compromised motor skills.
Aside from being the very first cannabis-based medicine to be approved in the U.S., Epidiolex is also the first-ever FDA-approved drug that treats Dravet syndrome.
FDA advisory panel’s earlier recommendation
It can be recalled that an FDA panel in April recommended unanimously that Epidiolex be approved for medical use. The panel, which is called the Peripheral and Central Nervous System Drugs Advisory Committee, had not expressed any concerns with regard to the drug’s safety or effectiveness.
And since the FDA is known to customarily follow its panels’ advice, many had already expected the agency to approve the sale and distribution of Epidiolex in the U.S.
The approval, according to FDA Commissioner Scott Gottlieb in a statement, serves as a reminder that the advancement of sound development programs that evaluate active compounds found in cannabis can lead to relevant medical therapies. He added that controlled clinical trials that test the efficacy and safety of a drug, coupled with careful review under the FDA’s drug approval process, are the most appropriate way to bring cannabis-based treatments to patients.
Epidiolex, CBD, and the DEA
Epidiolex contains highly purified cannabidiol or CBD, which is an active chemical ingredient in cannabis that does not possess psychoactive properties and does not create a high. CBD is just one of the more than 80 active components in cannabis.
However, CBD is still classified by the Drug Enforcement Administration as a Schedule I substance. This classification means that CBD is not considered to have any accepted medical benefits and that it is has a high potential for abuse.
This also means that even with the FDA’s approval, Epidiolex will not be readily available to patients who need it until the DEA reclassifies CBD.
The good news, though, is that the DEA is expected to reschedule CBD within 90 days, according to GW Pharmaceuticals.
GW Pharmaceuticals CEO Justin Gover called the FDA’s approval as a “historic milestone.”
Patients will soon have access to a cannabis-based medicine that has been studied thoroughly in clinical trials, has been manufactured with the assurance of quality and consistency, and will be available by prescription, Gover said.
Gover told CNN that Epidiolex will become available in the fall, but he would not give any information pertaining to its cost. He only said that pricing will still be discussed with insurance companies and that they will announce it later.
More treatable conditions may follow
A phase three clinical trial is currently underway for a third medical condition that may be treated by Epidiolex. This condition is called tuberous sclerosis complex, which is also characterized by seizures and begins in infancy. This condition causes a sudden stiffening of the body and the extremities, with the head bent forward.
According to Gover, if the results are positive, GW Pharmaceuticals will apply for the supplemental approval of this condition.