FDA Approves GW Pharmaceutical’s Cannabis Drug Epidiolex, Rescheduling Expected In 90 Days

The U.S. Food and Drug Administration (FDA) approved GW Pharmaceuticals’ (GWPH) cannabidiol (CBD) drug Epidiolex as an oral solution for the treatment of seizures associated with two rare and severe forms of epilepsy, Lennox-Gastaut syndrome, and Dravet syndrome, in patients two years of age and older. According to the press release, this is the first FDA-approved drug that contains a purified drug substance derived from marijuana and it is also the first FDA approval of a drug for the treatment of patients with the Dravet syndrome.

“Today, the FDA approved a purified form of the drug cannabidiol (CBD). This is one of more than 80 active chemicals in marijuana. The new product was approved to treat seizures associated with two rare, severe forms of epilepsy in patients two years of age and older,” said FDA Commission Scott Gottlieb, M.D.

He went on to add, “But it’s also important to note that 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.”

Gottlieb cautioned though that he remains concerned about the proliferation and illegal marketing of unapproved CBD-containing products with unproven medical claims. The FDA has taken action against companies making unsupported CBD claims in the past couple of years and the agency said it would continue to do so.

GW Says Rescheduling Within 90 Days

Even though the FDA has now approved the use of Epidiolex as a real treatment for these diseases, it doesn’t immediately change the status of cannabis as a controlled substance. The two defining factors of the schedule one designation include not having a medicinal purpose and being a highly addictive drug.

The FDA release stated, “Under the Controlled Substances Act (CSA), CBD is currently a Schedule I substance because it is a chemical component of the cannabis plant. In support of this application, the company conducted nonclinical and clinical studies to assess the abuse potential of CBD. The FDA prepares and transmits, through the U.S. Department of Health and Human Services, a medical and scientific analysis of substances subject to scheduling, like CBD, and provides recommendations to the Drug Enforcement Administration (DEA) regarding controls under the CSA. DEA is required to make a scheduling determination.”

The FDA did not say in its release whether it had delivered a recommendation to the DEA regarding Epidiolex and its schedule status.

However, in GW Pharmaceutical’s statement, the company said it would have to be rescheduled before it could be made available to patients. “Rescheduling is expected to occur within 90 days. Access is expected to be similar to other branded AEDs and EPIDIOLEX is expected to be available to appropriate patients by Fall 2018.”

“While we applaud the FDA for confirming what many of us have long known–that the benefits of cannabis are immense–we do find it a bit comical that the federal government now contradicts itself once again,” said Chris Driessen, President of Organa Brands U.S. “While the FDA touts the benefits of this new drug, the DEA maintains a Schedule One classification for cannabis, stating that it has no medical application and a high potential for abuse. We have to ask–does the left hand know what the right hand is doing?

Management Comments

“Today’s approval of EPIDIOLEX is a historic milestone, offering patients and their families the first and only FDA-approved CBD medicine to treat two severe, childhood-onset epilepsies,” said Justin Gover, GW’s Chief Executive Officer. “This approval is the culmination of GW’s many years of partnership with patients, their families, and physicians in the epilepsy community to develop a much needed, novel medicine. These patients deserve and will soon have access to a cannabinoid medicine that has been thoroughly studied in clinical trials, manufactured to assure quality and consistency, and available by prescription under a physician’s care.”

“In my practice, I often see patients with these highly treatment-resistant epilepsies who have tried and failed existing therapies and are asking about CBD,” said Orrin Devinsky, M.D., of NYU Langone Health’s Comprehensive Epilepsy Center and a lead investigator in the EPIDIOLEX clinical program. “I am delighted that my physician colleagues and I will now have the option of a prescription cannabidiol that has undergone the rigor of controlled trials and been approved by the FDA to treat both children and adults.”

“For those living with intractable seizures caused by LGS and Dravet syndrome, EPIDIOLEX represents a true medical advancement,” said Philip Gattone, president and CEO of the Epilepsy Foundation.  “Clinical development for these rare and severe conditions is essential, and today’s news brings hope for these patients and their families that a new treatment option may have the potential to help better control their seizures.”

Epidiolex

Epidiolex’s effectiveness was studied in three randomized, double-blind, placebo-controlled clinical trials involving 516 patients with either Lennox-Gastaut syndrome or Dravet syndrome. Epidiolex, taken along with other medications, was shown to be effective in reducing the frequency of seizures when compared with placebo. It uses CBD, which is a chemical component of the Cannabis sativa plant. CBD does not give patients a psychotropic effect like getting “high” that comes from the other part of the cannabis plant –  tetrahydrocannabinol (THC).