WASHINGTON — The FDA approved the first drug with an active ingredient derived from marijuana to treat rare, severe forms of epilepsy.
Cannabidiol (Epidiolex), an oral solution to treat seizures in Lennox-Gastaut Syndrome (LGS) and Dravet syndrome, was approved for patients two years of age and older.
“The FDA approval for cannabidiol is a landmark in American medicine,” said Orrin Devinsky, MD, of New York University Langone Health, who helped lead the product’s major clinical trials.
“This is the first time the government has approved a compound derived from the cannabis plant to treat any disorder — in this case, severe epilepsy due to Dravet and Lennox Gastaut syndromes. This should allow patients to have access to consistent cannabidiol doses made to the highest quality standards.”
Cannabidiol (CBD) is the first FDA-approved drug derived from marijuana and the first FDA-approved drug to treat patients with Dravet syndrome. It is a chemical component of the Cannabis sativa plant and does not cause the intoxication or euphoria that comes from tetrahydrocannabinol (THC). Epidiolex is a pharmaceutical-grade formulation; the approval was granted to GW Research Ltd.
LGS and Dravet syndrome are rare, severe, refractory epilepsy syndromes that emerge early in childhood. Both are linked to higher rates of mortality, primarily due to status epilepticus and sudden unexpected death in epilepsy patients (SUDEP).
An FDA advisory committee in April had recommended unanimously that Epidiolex be approved.
Three clinical trials of cannabidiol in LGS and Dravet syndrome demonstrated clinically meaningful and statistically significant reductions in seizure frequency.
“This is an important medical advance,” FDA Commissioner Scott Gottlieb, MD, said in a statement. “But it’s also important to note that this is not an approval of marijuana or all of its components. This is the approval of one specific CBD medication for a specific use. And it was based on well-controlled clinical trials evaluating the use of this compound in the treatment of a specific condition.
“Moreover, this is a purified form of CBD. It’s being delivered to patients in a reliable dosage form and through a reproducible route of delivery to ensure that patients derive the anticipated benefits. This is how sound medical science is advanced.”
Cannabidiol is structurally unrelated to other anti-seizure medications. Its anticonvulsant mechanism is unknown but does not appear to involve cannabinoid receptors.
last updated 06.25.2018