For the first time, the U.S. Food and Drug Administration has approved a cannabis-based drug. The drug, Epidiolex, has been approved to treat two types of epileptic syndromes. The drug’s approval comes as an increasing number of states have approved medicinal and recreational marijuana use. Epidiolex was recommended for approval by an advisory committee in April, and the agency had until this week to make a decision.
The twice-daily oral solution is approved for use in patients 2 and older to treat two types of epileptic syndromes: Dravet syndrome, a rare genetic dysfunction of the brain that begins in the first year of life, and Lennox-Gastaut syndrome, a form of epilepsy with multiple types of seizures that begin in early childhood, usually between 3 and 5.
FDA Commissioner Dr. Scott Gottlieb said in a statement “This is an important medical advance 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.”
The drug is the “first pharmaceutical formulation of highly-purified, plant-based cannabidiol (CBD), a cannabinoid lacking the high associated with marijuana, and the first in a new category of anti-epileptic drugs,” according to a statement from GW Pharmaceuticals, the UK-based biopharmaceutical company that makes Epidiolex. Justin Gover, chief executive officer of GW Pharmaceuticals, described the approval in the statement as “a historic milestone.”
He added that the drug offers families “the first and only FDA-approved cannabidiol medicine to treat two severe, childhood-onset epilepsies.” “These patients deserve and will soon have access to a cannabinoid medicine that has been thoroughly studied in clinical trials, manufactured to assure quality and consistency, and available by prescription under a physician’s care,” Gover said. He said Epidiolex will become available in the fall would not give any information on cost, saying only that it will be discussed with insurance companies and announced later.
Cannabidiol is one of more than 80 active cannabinoid chemicals, yet unlike tetrahydrocannabinol, or THC, it does not produce a high. The FDA has approved synthetic versions of some cannabinoid chemicals found in the marijuana plant for other purposes, including cancer pain relief.
According to the Epilepsy Foundation, up to one-third of Americans who have epilepsy have found no therapies that will control their seizures. With this approval, Epidiolex could be a new option for those patients who have not responded to other treatments to control seizures.