The Recent Approval of Epidiolex And Its Implications

The drug, Epidiolex, made by UK-based GW Pharmaceuticals is a purified form of CBD extracted from the cannabis sativa plant. The medicine is specifically designed for two difficult to treat forms of childhood epilepsy. Although the FDA has previously approved other, synthesized cannabinoids such as Dronabinol (a synthetic version of THC), this is the first time that regulators acknowledged the medical benefits of a compound derived from the cannabis plant itself.

The approval of medicinal CBD derived from the cannabis plant by the FDA is remarkable. Cannabis, including its active compounds CBD and THC, had been illegal under US federal law for decades. For medical marijuana, the approval is a big step that will likely lead to increased interest in the potential medical applications of CBD and other compounds found in the marijuana plant. What’s more, the approval of CBD, a substance which had been listed as a Schedule I drug until now, means that it needs to re-categorized as either a Schedule 2 or 3 drug, which will bring it down from its status as an illegal substance.

CBD AS AN EFFECTIVE TREATMENT FOR EPILEPSY

To green-lighting Epidiolex, the FDA considered three large clinical trials where the researchers presented strong evidence that CBD can significantly reduce some of the worst symptoms of two of the hardest-to-treat forms of epilepsy, Lennox-Gastaut syndrome and Dravet syndrome.

These syndromes of the illness have a higher early mortality rate than other types of epilepsy and do not respond to many of the other drugs available. Epidiolex is an oral solution with a strawberry flavour that patients take twice a day. The drug should be available in Europe by mid-2019 if the European Medicines Agency approves it early next year.

Discussing the scientific merits of the new CBD-based drug at a public FDA meeting earlier this year, panel member and senior scientist John Mendelson at the Friends Research Institute called the drug^[1] “a clear breakthrough for an awful disease”.

Parents of children with epilepsy who discovered CBD as an effective treatment had previously been forced to violate the law. Now, the families of these patients, and with them researchers and cannabis advocates, are hailing the arrival of Epidiolex as a long-awaited treatment.

EPIDIOLEX VS. OTHER CBD PRODUCTS

Although the drug will soon be available by prescription in Europe and the US, many patients will likely turn to less expensive CBD alternatives, such as those available in marijuana dispensaries. However, researchers and advocates caution against this.

Their main argument is that it is impossible to verify what is contained in other CBD products on the market. Currently, CBD-based products such as CBD oils, tinctures and salves are already widely available in those states where the use of marijuana for medicinal purposes is legal.
Chief officer Laura Lubbers of the nonprofit Cure, a group that funds epilepsy research, points out that the difference between Epidiolex and other CBD products is that Epidiolex is well studied and well-controlled, but CBD products available in dispensaries are not. She is not wrong when she says this since recent scientific studies give merit to her rather cautions stance:

In a study published^[2] in the Journal of the American Medical Association in 2017, researchers tested 84 CBD products from 31 different online sellers. They found that 7 out of 10 products had different levels of CBD than what had been specified on the label. Half of the products examined in the study contained more CBD than what was indicated and a quarter of them contained less. In 18 of the samples tested, the researcher also found THC, despite the substance not being listed on the label.

The president of the American Epilepsy Society and professor of neurology and epidemiology at the Albert Einstein College of Medicine, Shlomo Shinnar, says that the important thing here is that the CBD approved by the FDA is pharmaceutical-grade CBD made under strict quality standards, just as other FDA-approved drugs. He underlines that this is not the same as when people obtain a different CBD product that merely vaguely specifies that it is high in CBD.

CAN EPIDIOLEX ALSO BE USED TO TREAT OTHER CONDITIONS?

Although Epidiolex is designed to treat two specific types of epilepsy only, medical professionals could technically prescribe it also for other conditions. Such an “off-label” prescription for Epidiolex by doctors would be similar to as when they prescribe drugs for other uses, such as when they prescribe the anesthetic ketamine for the treatment of PTSD and severe cases of depression. With Epidiolex soon legally available as a medicine, there is obviously big potential for such alternative uses of the drug beyond its intended purpose.

Advocates do expect that once Epidiolex has been approved it will likely also be tried for the treatment of other conditions. Could the approval of Epidiolex mean wider availability of CBD for patients with chronic pain, anxiety, and other conditions?

The vice president of investor relations for GW Pharmaceuticals, Stephen Shultz, cautions that an official approval of their drug for other uses will likely not happen any time soon. He explains how companies evaluate their medicines and its use for specific patient populations. This is because different patient populations and different therapeutic targets may respond differently to the same medicine. Epidiolex had been approved for treating epilepsy because of the clinical data backing its effectiveness and safety. No such clinical data exists yet for using it for other purposes. This is also why Greenwich Biosciences, the US subsidiary of the maker of Epidiolex, won’t promote the drug for any other uses outside of the Dravet and Lennox-Gastaut populations. Prescribing the medicine for other uses is then between patients and their physicians.

THE APPROVAL OF EPIDIOLEX MAY BENEFIT DEEPER RESEARCH INTO CANNABIS-DERIVED DRUGS

Despite Epidiolex for now only approved for these two specific forms of epilepsy, the approval will in all likelihood greatly benefit more research into cannabis-derived drugs. Green lighting CBD also means that the Drug Enforcement Agency has to reschedule CBD. Now, with the approval of Epidiolex, CBD is about to leave its official status as an illegal drug and will become a medicine, similar to Adderall. For the medicinal marijuana sector, this is without a doubt a big step in the right direction.

Advocates are looking forward to big positive changes that will affect CBD manufacturers and the medicinal marijuana industry as a whole. Finally, there seems to be a light at the end of the tunnel for an industry which operates in a legal grey area.

Researchers have now also begun to look into THC and other compounds found in cannabis for their potential medicinal uses. The recent approval of a synthetic form of THC for medicinal use is a good example for this. The approval of Epidiolex, the first drug made directly from a compound found in cannabis is without question no less than a milestone. We can hope that more positive changes for medicinal marijuana, including its users and uses will be on the horizon.