CBD-Based Drug Epidiolex Receives Green Light From FDA: A double-edged sword?

CBD-Based Drug Epidiolex Receives Green Light From FDA: A double-edged sword?

For the first time ever in history, the US Food and Drug Administration (FDA) is set to approve a CBD-based drug called Epidiolex.

Manufactured by UK-based GW Pharmaceuticals, Epidiolex has shown promising results in clinical trials for the treatment of pediatric epilepsy and seizures. CBD, cannabidiol, is the non-psychoactive compound in cannabis.

Last Thursday, an FDA panel endorsed Epidiolex with a unanimous 13-0 vote. Experts weighed in, saying that the CBD-based drug had significant benefits in treating Lenox-Gastaut Syndrome (LGDS), as well as Dravet Syndrome (DS), two rare forms of epilepsy. The FDA doesn’t always follow recommendations from advisers, although when the votes are overwhelmingly positive, these results are expected.

“This is clearly a breakthrough drug for an awful disease,” says Dr. John Mendelson, one of the panelists present in the advisory committee meeting.

The approval of the drug was given a deadline for approval, which is set for the end of June. However, it’s possible for the approval to come well before the deadline since regulators told the advisors that Epidiolex would be placed on review much quicker.

According to GW Pharmaceuticals CEO Justin Gover, the green light from the FDA “will make a sea change in the acceptability of cannabinoids as therapy.”

Should Epidiolex get the seal of approval from the FDA, it will be among the first cannabinoid-based drug that has been derived from the actual cannabis plant instead of synthetic cannabinoids that doctors can finally prescribe to their patients. Currently, there are already other FDA-approved drugs on the market that have been manufactured derived from synthetic THC (tetrahydrocannabinol), the psychoactive compound in cannabis, as well as other compounds found in the plant. These drugs are used to alleviate nausea, vomiting, and appetite loss among cancer and HIV/AIDS patients.

“Although the review is still ongoing, the risk-benefit profile established by the data in the application appears to support approval of cannabidiol for the treatment of seizures associated with LGS [Lennox-Gastaut syndrome] and DS [DravetS Syndrome], the FDA said in a report. “The statistically significant and clinically meaningful results from these three studies provide substantial evidence of the effectiveness of CBD for the treatment of seizures associated with LGS and DS,” adds the FDA.

Advocates pushing for cannabis-based treatments, especially for the treatment of epilepsy, were happy with the outcome from the panel.

“This is a very good development, and it basically underscores that there are medicinal properties to some of the cannabinoids,” says Dr. Igor Grant, the director of the Center for Medicinal Cannabis Research at the University of California San Diego. “I think there could well be other cannabinoids that are of therapeutic use, but there is just not enough research on them to say.”

Lennox-Gastaut syndrome affects around 30,000 children as well as adults, but the number is much less for those suffering from Dravet syndrome. Since these conditions are extremely rare, GW Pharmaceuticals was able to obtain an orphan drug status for Epidiolex.

“It’s very important that we have additional treatments because these patients have very, very difficult to control seizures,” says University of Alabama at Birmingham neurology professor Dr. Jerzy P. Szaflarski, who also oversees the epilepsy division at the university. “I get questions about cannabidiol almost every day.”

Potentially Serious Side Effects

Unlike whole plant medicine, which has no side effects, Epidiolex comes with potential unwanted consequences – which was indicated on the briefing papers prepared by the FDA.

Liver damage.

However, according to panelists, liver damage may be managed if doctors monitor patients for any warning signs. Other common potential side effects from Epidiolex include sleep problems, fatigue, vomiting, and diarrhea.

If the FDA approves Epidiolex, this means that cannabis should be rescheduled. How can it still be considered a Schedule 1 substance if the FDA approves a cannabis-based drug already?

But still, this doesn’t mean that a CBD-based pharmaceutical medicine is still better than whole plant medicine.

During earlier trials of Epidiolex, 12 patients chose to discontinue the treatment because of side effects. One of the participants of the study also died, although of course GW Pharma says that it isn’t related to Epidiolex. In the same study, 74% of the patients reported experiencing an “adverse event”.

The FDA approval of Epidiolex may just be a double-edged sword.