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Bottles of Epidiolex are seen at a production facility. Epidiolex is a tincture derived from marijuana plants and FDA approved to treat seizures. (Provided by Greenwich Biosciences)

In Dr. Susan Koh’s clinic at Children’s Hospital Colorado, patients earlier this year anxiously awaited the approval of a new drug to treat two rare seizure disorders.

The conditions — one called Dravet syndrome and another called Lennox-Gastaut syndrome — often take hold when kids are still in the crib. The disorders are so difficult to manage that most patients with them have tried and failed multiple other anti-seizure drugs. And the new drug, called Epidiolex, wasn’t just any other coming through the pipeline. It was the world’s first pharmaceutical drug derived from naturally grown cannabis.

That meant it held a special mystique in Colorado, where the promise five years ago of marijuana-derived treatments sold through the state’s medical marijuana industry drew hundreds of families from across the country. But, by gaining approval from the federal Food and Drug Administration, Epidiolex had some things none of those artisanal treatments could offer — peer-reviewed clinical trials arguing for its efficacy and full-fledged federal legality.

So, when Epidiolex was officially OK’d this summer and made available for prescription this fall, Koh said she began writing scripts. Weeks later, though, most of her patients haven’t received their medicine.

“The process actually is quite complex and long,” said Koh, a neurologist who specializes in treating epilepsy.

Koh said Epidiolex’s maker, the British drug company GW Pharmaceuticals, has thus far made the medicine available only through five subspecialty pharmacies around the country. Rather than being able to fill the prescription at any Walgreens, Koh said prescriptions first go through a central hub before being sent out to the pharmacies, which deliver the medication via the mail.

Cannabis plants used to make Epidiolex are grown in greenhouses in southern England. Epidiolex is an FDA-approved drug used to treat rare seizure disorders. (Provided by Greenwich Biosciences)

On a website developed specifically to provide information on Epidiolex, Greenwich Biosciences, an arm of GW, says the pharmacies verify “insurance eligibility and other requirements” and then set up a phone call with a patient’s family to go over dosing instructions before sending the medicine.

A spokesman for GW did not respond to a request for comment.

Epidiolex comes as a tincture that is squirted into a patient’s mouth with a syringe — much like the artisanal cannabis oils many patients already use. According to the FDA, it is pronounced EH-peh-DYE-oh-lex.

The active ingredient in both Epidiolex and the artisanal products is cannabidiol, or CBD, a non-psychoactive compound found in marijuana. In studies, Epidiolex showed few serious side effects.

Koh said several of her patients and their families are hoping that Epidiolex will provide more consistency batch-to-batch and clearer instructions for dosing. Artisanal CBD producers are largely unregulated, creating a wide variety of production methods and product quality. With the approval of Epidiolex, the FDA may also start cracking down more on artisanal CBD producers.

“I think the hype has fallen off a little bit,” Koh said of CBD use by her patients. “But we still do see a lot of kids who are on CBD from a medical marijuana dispensary.”

Another hurdle facing patients who want to use Epidiolex is the cost. Koh said insurance typically covers the costs if Epidiolex is prescribed for Dravet and Lennox-Gastaut. But it’s possible that insurers would deny coverage if Epidiolex were prescribed off-label for a different seizure disorder, leaving patients to pick up a bill in the thousands of dollars.

Greenwich Biosciences says on its website that it runs a program to help low-income patients afford the medicine.

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Colorado’s Medicaid system also covers Epidiolex, though it is too soon to know how many people in the Medicaid system have been prescribed it, said Marc Williams, a spokesman for the Colorado Department of Health Care Policy and Financing, which administers Medicaid in the state.

In order for the drug to be covered by Medicaid in Colorado, doctors must first seek pre-approval from state officials — as is the case with many drugs. But there is no requirement that patients have tried and failed other medications first.

Koh said she will typically prescribe Epidiolex as part of a suite of anti-seizure medicines and not as the only anticonvulsant drug that the patient is on. She said she also wants patients to try other, more established medications first.

“It’s like a third- or fourth- or fifth-line drug,” she said. “Those kids have tried and failed a lot of medications.”

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John Ingold is a co-founder of The Colorado Sun and a reporter currently specializing in health care coverage. Born and raised in Colorado Springs, John spent 18 years working at The Denver Post. Prior to that, he held internships at...