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July 11, 2018

After FDA approves first drug made from marijuana, what happens next?

By Leigh Hopper

Marc Fuya/flickr

On June 25, the U.S. Food and Drug Administration approved the first drug made from marijuana – Epidiolex -- to treat two rare and devastating forms of epilepsy, Dravet syndrome and Lennox-Gastaut syndrome.

Dr. Shaun Hussain, a pediatric neurologist at UCLA who treats children with epilepsy, believes the medication will be a game-changer for many patients.

“This is huge,” Hussain said. “The cognitive consequences of these diseases are severe. A lot of these kids aren’t in regular school, they’re not getting jobs, they’re not growing up to be independent. So there’s a tremendous unmet need for effective treatment.”

We asked Hussain a few questions about the new drug.

Dr. Shaun Hussain

What is Epidiolex?

Cannabis produces two main “cannabinoids” known as THC and CBD. THC is the compound that makes you “high,” gives you the munchies and fights nausea. CBD, or cannabidiol,  which doesn’t make you high, is the active ingredient in Epidiolex. It’s extracted from a proprietary strain of marijuana, mixed in an oil, and taken twice daily by mouth.

The FDA-approved drug contains almost pure CBD, with only trace amounts of other cannabinoids. This is different from the varieties of non-prescription CBD oil you might find at a medical marijuana dispensary. These contain numerous other cannabinoids, including at least small amounts of THC.

How does it work?

In clinical trials, Epidiolex was shown to reduce the number of seizures by about 40 percent in patients with Dravet or Lennox-Gastaut syndrome. That is significant, because these diseases start in early childhood and causes countless seizures, which can interfere with normal neurodevelopment. In those trials, some of the drug’s side effects were sedation, elevated liver enzymes and diarrhea.

It’s unclear exactly how Epidiolex works. A few novel receptors have been identified in the brain that may mediate the actions of cannabidiol. I would note that the No. 1 epilepsy drug in the U.S., levetiracetam, acts via largely unknown mechanisms as well. This phenomenon, where drugs work but we don’t know exactly why, is common among epilepsy treatments.

How much will it cost?

It will cost in the thousands per month. Presumably, insurance companies will pay.  For example, another recently-approved drug for Lennox-Gastaut syndrome costs $5,000 to $10,000 a month.

Some parents were already giving their children CBD oil that they bought from other sources; will they switch?

Parents trying to help their children with CBD oil have been spending about $400 to $2,000 a month. That’s cheaper than Epidiolex, but it’s not covered by insurance. And in many cases, it’s not helping very much. Now, is that because CBD won’t help their child and Epidiolex is unlikely to help as well? Or will Epidiolex, with greater consistency and dosage, yield better results? In clinical trials, Epidiolex was given at a dosage of 20 to 40 milligrams per kilogram of body weight per day. That dosage is about 10 times higher than how people use non-prescription CBD oil.

Are we likely to see the FDA approve more cannabis-derived drugs?

It is a pivotal moment. We're going from the position that marijuana is only harmful, that it’s a ‘gateway drug,’ to the belief that cannabis actually has some real, therapeutic potential. That said, products that fall under the umbrella of medical marijuana will still face challenges from regulators. For example, forms of medical marijuana with high CBD concentrations that most resemble Epidiolex, and which inspired its development, are illegal under federal law. To date, legislators on both sides of the aisle have been reluctant to change the regulation and legal standing of marijuana. We shall see if Epidiolex’s approval leads to regulatory changes.

 

Tags: News & Insights, Research

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