An explanatory of a new drug drug based on marijuana approved by the FDA

Epidiolex, a cannabidiol-based (CBD) drug derived from the marijuana plant has been approved by the Food and Drug Administration (FDA) for children and adults over 2 years of age. It deals with Dravet syndrome and Lennox-Gastaut syndrome), two rare epilepsies that mainly affect children.

What is Epidiolex?

Epidiolex is an oral liquid-based medicine made from cannabis cannabidiol (CBD). Compared with the tetrahydrocannabinol (THC) portion, CBD is not psychoactive, so taking this medication would not result in a high level for the user.

CBD acts on different parts of the nervous system compared to THC. According to the Centers for Disease and Control (CDC), THC and CBD can both be useful for nausea caused by cancer treatments. However, none of the substances derived from marijuana has been approved medically since they both fall under the Schedule 1 drug classification, WHAT is traditional knowledge, according to the Drug Enforcement Administration. (DEA).

Kathy Young / AP FILE

Why has it been approved?

The drug was approved by the FDA on Monday after a review of four major studies that examined Epidiolex for the treatment of Lennox-Gastaut syndrome and Dravet syndrome in adults and children.

The first major study examined the effect of Epidiolex for Lennox-Gastaut syndrome was published in the New England Journal of Medicine in May. In the study, the researchers recruited 225 patients, ages 2 to 55 years (all of whom had two or more seizures a day) at two different doses of Epidiolex or a placebo that contained no medication. The trial lasted 14 weeks. Mean seizure frequency dropped 41.9% in the highest dose group and 37.2% in the lowest dose group and 17.2% in the placebo group (placebo inactive often has a medicinal effect).

"We knew the potential of CBD in the treatment of epileptic seizures for years, so we expected an answer, yet we were surprised at the robustness of the response in this study," said Dr. Anup Patel, neurologist. Pediatric at Nationwide Hospital Children's Hospital of Ohio and one of the leading authors of the study, at ABC News.

The second major study was published in the New England Journal of Medicine in May, and it examined the effect of Epidiolex for Dravet syndrome. Researchers recruited 120 children and young adults to compare Epidiolex with standard antiepileptic therapy. After 14 weeks, the average frequency of seizures decreased by at least half in 43% of people taking Epidiolex and in 5% of people taking the drug without epilepsy.

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Dravet syndrome and Lennox-Gastaut syndrome are diagnosed by a clinical and EEG examination by a physician, ideally a doctor specializing in childhood brain diseases, known as a pediatric neurologist. Dravet's syndrome is confirmed by a genetic test.

What are the side effects of this medicine?

In the trial, the main side effect was hepatic toxicity, which appeared as an increase in liver enzymes.

"Other people may have experienced diarrhea, vomiting or vomiting that was more muffled, nauseated or experienced, but these symptoms did not limit life," Patel told ABC News.

Dr. Nicholas Chadi, a pediatrician who is completing his fellowship at the Boston Children's Hospital in Pediatric Addictions and Adolescents, points to limited research on CBD side effects in general.

"It's only recently that we've been able to separate CBD from THC, and create substances with very high CBD content," Chadi told ABC News. "So, we do not really know much about the effect of CBD on the developing brain or whether it could create a long-term addiction."

Chadi noted that there was significant research on the negative implications of THC, which is what produces the top of "weed" and affects the developing brain.

"There is research that indicates that CBD could interact with other drugs such as statins, and increase the amount of these drugs in the blood," Chadi said.

Andrew Harnik / AP FILE

When can parents of children with LGS or Dravet syndrome have access to Epidiolex?

The drug must undergo several steps before it is available on prescription. First, the DEA will be heading towards unsubscribing CBD as a Schedule 1 drug. The pharmaceutical manufacturer behind the drug, GW Pharmaceuticals, is negotiating prices with health insurance companies. The process is expected to take 90 days, according to the FDA's press briefing on Monday. ABC News also confirmed this planned deadline with Christy Curran, spokesperson for GW Pharmaceuticals.

"We do not expect to know the price before the fall, but over the next few weeks we will work to establish a price with the insurance providers.We think it will be covered by most insurers, "said ABC News Justin Gover, chief executive of GW Pharmaceuticals.

Can it be used for other epilepsy conditions in children and adults?

Not currently.

"The [pharmaceutical company] is also looking for other conditions for which the drug may help, but we still do not know what they are, "Patel told ABC News, adding that the approval of the FDA would facilitate the study of the drug by other groups. .

What should parents know now?

All parents should be aware of unregulated CBD products on the market, a problem raised by the FDA and Patel.

"We receive at least one request a day from patients who use or plan to use CBD that they have ordered online. The problem is that these substances are not regulated, so it is difficult to know how to dose them. They can also be contaminated by other things, so safety, especially for children, is a concern, "Patel told ABC News.

"Patients should have conversations with their doctors about these products," warned FDA Commissioner Scott Gottlieb, MD "As to whether this product provides them with the treatment they are looking for. Have not been tested for their safety and effectiveness. "

Overall, approval is great news for parents of children with Dravet syndrome or LGS, although it will take at least three months before the drug enters the pharmacy. .

"We encourage our patients to wait and explain to them why it's a better way to go instead of using things that have not been studied or tested," Patel said. "There is light at the end of our tunnel. can prescribe the drug for all patients in need. "

Amitha Kalaichandran is a pediatric resident at the University of Ottawa and works at ABC News Medical Unit in New York.