Regulators at the US Food and Drug Administration today granted market approval for Epidiolex, a prescription medicine containing a standardized formulation of plant-derived cannabidiol (CBD), for the explicit treatment of two rare forms of severe epilepsy: Lennox-Gastaut syndrome and Dravet syndrome.
With the historic approval, the London-based company behind the drug, GW Pharmaceuticals plc, is expecting another consequential decision in the coming weeks: getting the US Drug Enforcement Administration to reclassify CBD. It's not yet clear why the ingredient, known as CBD, reduces seizures in some people.
Critically, the drug will be available to patients nationwide, not just in states where cannabis is legal for medical purposes. 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 that support appropriate dosing needed for treating patients with these complex and serious epilepsy syndromes.
Side effects with the drug include diarrhea, vomiting, fatigue and sleep problems.
Two months after a unanimous recommendation by an outside group of FDA experts and some glowing recommendations in the internal review, GW will now set out to open up a new class of meds after demonstrating the drug's ability to prevent seizures in children suffering from rare cases of Lennox-Gastaut syndrome and Dravet syndrome.
He added: "We anticipated that Epidiolex will be the first of many potential FDA-approved medicines based on the cannabis plant". And like other drugs that treat epilepsy, Epidiolex will come with a special warning that use may be tied to a higher odds of depression, aggression and suicidal thoughts. Once the FDA approves Epidiolex, the DEA will be compelled to deschedule it from a Schedule I drug on the Controlled Substances Act (defined as "no recognized medical use") to either a Schedule II or III drug.
The FDA previously issued warnings to CBD producers that claimed their products could treat specific diseases, such as cancer or Alzheimer's. Before the panel meeting, FDA staff also backed the drug in their own report, saying the company offered "substantial evidence" of efficacy.
GW Pharmaceuticals grows the plants in the United Kingdom.
Still, Jackson is glad that her family's experience could now provide new treatment options for other childhood seizure sufferers.
She says 17 doctor-prescribed drugs didn't help her son.
Nine states plus Washington, D.C., have legalized medical marijuana, and the FDA said even more are considering it, particularly after it approved Epidolex. This status has stifled rigorous research, because the federal government will not pay for it. A bill was introduced to the House a year ago to legalize the use of marijuana, but it has yet to reach the floor for vote.
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. And the company plans to continue importing the medicine, bypassing onerous US regulations on manufacturing restricted substances.