June 26, 2018
By: medical marijuana law attorney, Richard M. Blau, Chair, Medical Marijuana Practice Group
The U.S. Food and Drug Administration on Monday approved the country’s first drug derived from marijuana, a medication that treats two rare and devastating forms of epilepsy. The drug, produced by GW Pharmaceuticals plc (Nasdaq: GWPH) and called Epidiolex®, is made from cannabidiol (CBD), a component of marijuana that does not give users a high. It is formulated as an oil, and in clinical trials was shown to reduce seizures associated with Dravet or Lennox-Gastaut syndrome by approximately forty-percent (40%).
Patients with Lennox-Gastaut and Dravet syndromes, which typically emerge in the first few years of life, can suffer from debilitating and recurrent seizures, sometimes dozens a day. One in five patients is estimated to die before they are 20 years old. There are six other drugs approved to treat seizures associated with Lennox-Gastaut, but none approved for Dravet.
In announcing the approval, FDA Commissioner Scott Gottlieb said that the agency’s action "serves as a reminder that advancing sound development programs that properly evaluate active ingredients contained in marijuana can lead to important medical therapies." He also reaffirmed that "the FDA is committed to this kind of careful scientific research and drug development."
As noted in a prior posting on this Website, an FDA Advisory Committee issued the first ever positive recommendation for a plant-based pharmaceutical cannabidiol product in April of 2018, when the committee recommended approval of the investigational cannabidiol oral solution known as Epidiolex® to treat seizures in patients with severe forms of epilepsy. While some concerns about the drug’s effects on the liver were noted, regulators have concluded that such risks can be contained by doctors through careful patient monitoring during treatment.
Commissioner Gottlieb also used the approval to highlight GW’s long-term commitment to the clinical development of Epidiolex as a model for bringing products made from marijuana to the market. The FDA granted "Priority Review" designation for GW’s application. The fast-track designation was granted for Dravet syndrome. Orphan Drug designation was granted for both the Dravet syndrome and Lennox-Gastaut syndrome indications. Nevertheless, the Epidiolex approval process took many years. Still, the FDA maintains it is willing to help companies that want to pursue such research programs in order to develop data from clinical trials that demonstrate their products work and are safe.
However, the FDA’s position is in contrast to the current stance against cannabis and marijuana legalization taken by the Trump Administration and its Department of Justice, led by Attorney General Jefferson Sessions, III. Despite FDA approval, GW cannot begin marketing Epidiolex in the United States until the Drug Enforcement Administration (DEA) reclassifies CBD so that it is no longer a Schedule 1 narcotic under the Controlled Substances Act (CSA). Schedule 1 drugs are deemed to have no medical value and present a high risk of abuse. Marijuana has been listed as a Schedule 1 drug for generations. Under the CSA, CBD is currently a Schedule I substance because it is a chemical component of the cannabis plant.
Although Epidiolex was only approved for the two specific conditions, analysts expect doctors to prescribe it off-label for a variety of epileptic diseases. It comes from a proprietary strain of cannabis grown by GW that has been bred to have high levels of CBD and low levels of tetrahydrocannabinol (THC), the cannabis constituent that produces the euphoric high typically associated with marijuana and cannabis-derived products.
In the FDA’s announcement, Commissioner Gottlieb expressly cautioned that the agency’s approval of Epidiolex should not be construed as opening the floodgates for marijuana-based pharmaceuticals:
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."
The FDA previously has issued warning letters to companies that promoted their CBD products as cancer or Alzheimer’s fighters. Interestingly, the warning focused not on the products’ CBD content as violative of the CSA, but instead on the unproven and potentially fraudulent nature of the product’s marketing representations. As noted in the FDA’s announcement approving Epidiolex, "[m]arketing unapproved products, with uncertain dosages and formulations, can keep patients from accessing appropriate, recognized therapies to treat serious and even fatal diseases."