Will Epidiolex Become the First Ever FDA Approved Cannabis-Based Drug?
May 2, 2018

Will Epidiolex Become the First Ever FDA Approved Cannabis-Based Drug?

MarijuanaBreak Staff MarijuanaBreak Staff / Updated on May 2, 2018


Some very “exciting” news has recently hit the marijuana world. You might even call it an iconic movement. For anyone of you who may have been living under a rock for the past few days, or perhaps you don’t keep up to date with the latest news, the US Food and Drug Administration (FDA) has for the first time recommended the approval of a drug derived from cannabis. The drug in question, as many of you are probably wondering, is Epidiolex. The product is a ‘pure CBD’ plant extract, developed by British company GW Pharmaceuticals.

On the one hand, this is a vital step in acknowledging that cannabis has a valid medical use, and will hopefully loosen the research restrictions that have been keeping scientists from figuring out the numerous health benefits of cannabis. On the other hand, though, it means more money for the massive pharmaceutical industry – which may not be such a bad thing if the drug ends up helping millions worldwide.

Earlier in April, the FDA committee unanimously recommended the approval of Epidiolex, which would be the first plant-derived cannabidiol medicine available for prescription use in the United States. The final vote deciding on whether or not to approve the drug will take place in June.

Now, as we are sure that most of you will be as intrigued as we are over this recent development in the world of cannabis, in this article we will take a closer look at Epidolex, the company behind it, what they do, and why the drug is in the process of being approved.

What is Epidiolex

Epidiolex is a liquid formulation of plant-derived Cannabidiol (CBD), manufactured by a company called GW Pharmaceuticals. It’s used to treat severe forms of epilepsy including Dravet syndrome and Lennox-Gastaut syndrome, the former being a rare genetic dysfunction of the brain that begins the first year of life, and the latter being a type of epilepsy with multiple forms of seizures. It’s also used to treat infantile spasms and tuberous sclerosis complex, both of which start in infancy and cause a sudden stiffening of the arms, legs, and body with the head bent forward.

The FDA panel has recommended the approval of the drug to treat both Dravet syndrome and Lennox-Gastaut syndrome. These are historically among the most difficult forms of epilepsy to treat, with the majority of patients continuing to have seizures despite using the medications that are currently available on the market. There are approximately 30,000 adults and children who suffer from Lennox-Gastaut syndrome, and even fewer than that with Dravet syndrome. These numbers make this a rare condition, which is why Epidiolex has received what’s known as an “orphan drug designation.” This means that the drug has been developed specifically to treat a rare medical condition. To qualify for orphan drug designation, both the drug and disease must meet certain criteria specified by the ODA and FDA.

The drug is available in the form of a viscous liquid to be dispensed in syringe droppers, and is non-psychoactive. In other words, it doesn’t give users the effects of being “high.” Also, Epidiolex is made up of 98% CBD with zero THC, and the remainder consists of trace amounts of other cannabinoids.

The move towards the approval of Epidiolex is a great development for the cannabis world, and specifically cannabis researchers. It basically admits that there are in fact valid medical properties to some of the cannabinoids found in weed. Since marijuana is still considered an illegal Schedule I drug at the federal level, though, (along with the likes of heroin, cocaine, and LSD), researchers had to go through a rather complicated bureaucratic process to conduct their study on Epidiolex.

If approved, Epidiolex will be the first in a new category of anti-epileptic drugs. Various studies have been conducted on the drug, each of which showed a significant reduction in monthly convulsive seizure frequency. GW is currently evaluating additional clinical development programs in other orphan seizure disorders, that will be presented prior to the final approval voting in June.

Who is GW Pharmaceuticals

GW Pharmaceuticals are one of the world’s leaders in the development of plant-derived cannabinoid therapeutics. Founded in 1998, the company is listed publicly on the NASDAQ Global Market (GWPH), and it has operations in both the UK and the USA. There are two well-known entrepreneurs behind the company, Dr. Geoffrey Guy and Dr. Brian Whittle, both of whom are from the UK biotech sector. They worked closely with the UK’s Medicines Regulatory Authority and the UK Home Office to set up licensing for the company, and to establish the necessary procedures to facilitate the progress of GW’s cannabinoid research program.

The first clinical trials conducted by GW Pharmaceuticals evaluated the different cannabinoid formulations as potential therapeutics. Their initial focus was on the development of Sativex, which is an oral mucosal spray that consists of two principal cannabinoid compounds – CBD and THC. Sativex has been studied in more than 20 randomized placebo-controlled trials (with over 3,000 patients) and has been the subject of many regulatory submissions and approvals.

In 2010, GW Pharmaceuticals made history when they launched the world’s first prescription cannabis-derived drug. The product was of course none other than Sativex, which was produced to treat multiple sclerosis. To date, it has been approved for treatment in numerous countries outside of the United States. Their latest project, though, is of course Epidiolex, which studies have shown to be highly effective in treating children with the deadly forms of epilepsy previously mentioned.

As stated on their website, the mission of GW Pharmaceuticals is to “unlock the potential of cannabinoid medicines,” with the purpose of “addressing serious medical conditions [that have] limited treatment options.” The Company has a deep pipeline of additional cannabinoid-based projects, which includes compounds that are currently in Phase I and II trials for the treatment of schizophrenia, epilepsy, and glioma, which involves tumors of the nervous system.

How does Epidiolex work?

As we speak, intense research is still being done on epidiolex and cannabidiol to determine more about why they are an effective substance used to treat seizures. On the GW website, they explain that CBD and Epidiolex can reduce inflammation and physical stress in the brain, which leads to the reduced likelihood of experiencing a seizure.

However, the company does confirm that “the precise mechanism of action of the anti-epileptic cannabinoids is [still] a subject of intensive ongoing research at GW Pharmaceuticals.” In other words, they are saying that they still have a lot of work to do before they fully understand the complex physiological mechanisms involved with how Epidiolex works.

Numerous studies have already been funded by GW Pharmaceuticals, some of which were undertaken in the U.S. using non-approved, ‘artisanal’ CBD preparations to control seizures. During these studies, 84% of parents reported a decline in the frequency of their child’s seizures, and 11% of these reported a complete cure of seizures. Moreover, 42% reported a more than 80% reduction in seizure frequency, and 32% reported a 25-60% reduction in seizures.

Systematic investigations that have explored the efficacy and safety of Epidiolex have also produced promising results for previously untreatable epileptic conditions. A 12-week clinical study that was done on Epidiolex, for example, found that a third of participants were seizure-free after undergoing treatment.

The fact is that current “traditional” treatments involve various mind-numbing medications, diets, brain surgeries, invasively implanted electrical stimulation devices, and alternative therapies that offer little to no relief of symptoms. Epidiolex, on the other hand, is offering parents and children an unprecedented hope.

Why the FDA is considering approval of Epidiolex

The biggest concern that was surrounding Epidiolex was the potential risk of liver injury, but the FDA official document notes that physicians can manage this by closely monitoring liver enzyme levels. Also, it was determined that the risks associated with CBD were acceptable, especially given the effectiveness of Epidiolex on both Dravet Syndrome and Lennox-Gastaut Syndrome, which are classified as “debilitating and life-threatening disorders.”

Another major point of consideration is the fact that Epidiolex is made up of 98% CBD, which is a non-psychoactive component and thus doesn’t have the same side effects that often associated with cannabis. (Among other things, these side-effects include THC-induced anxiety and paranoia).

The FDA also confirmed that GW Pharmaceuticals has submitted positive results of efficacy based on three randomized, double-blind, placebo-controlled trials that were conducted on patients with both Dravet Syndrome and Lennox-Gastaut Syndrome. The positive results of these studies provided substantial evidence of CBD being an effective treatment for seizures associated with both conditions.

Probably most noteworthy of all, however, is the fact that there is a serious lack of “traditional” medication currently available to treat these seizures. This has left desperate parents of children with epilepsy at their wit’s end, with most of the drugs currently on the market providing no effective treatment.

Laura Lubbers, for instance, who is the Chief Scientific Officer of a non-profit epilepsy research organization called CURE, recently told Business Insider that her group saw GW’s drug as a “long-awaited” treatment. This is especially true for patients who haven’t responded to other drugs. She went on to state that what makes Epidiolex most appealing is that it’s a “well-studied,” and “well-controlled” product.

Aside from GW’s own research, there have been additional studies conducted on Epidiolex’s anti-seizure effects. One clinical trial, for example, looked at the effects of the drug on 225 young people who suffered from Lennox-Gastaut syndrome. The participants were split into different groups, where they were given either a high dose, low dose, or a placebo treatment for 14 weeks. Results showed that the participants in the high-dose group saw a seizure frequency decrease of 42%, while those who were given the low-dose saw a reduction of just 37%. In comparison, those who were given the placebo only saw a 17% seizure frequency reduction.

Because GW Pharmaceuticals was able to show that their product addressed a critical need, they were able to apply for a designation to fast-track the FDA’s lengthy approval process. This goes to show that the FDA truly believes in Epidiolex and it’s potential benefits. No matter your opinion on the subject, or the less-than-appealing relationship between Big Pharma and the cannabis industry, it’s clearly a breakthrough treatment for what is currently considered a condition with no cure and very limited treatment options.

Final thoughts on the Potential FDA Approval of Epidiolex

The fact that Epidiolex received a unanimous vote of approval from an outside panel of advisors to the FDA is certainly a great sign for the final vote that’s set to take place in June. All things considered, it’s looking more and more likely that the drug will officially be approved soon.

It is worthwhile to mention, though, that approval would only be for a single compound (CBD) that comes from the cannabis plant, and it would only be applicable for the treatment of two specific forms of epilepsy. We don’t yet know what this approval will mean for other conditions, other cannabinoids found in cannabis, or for the overall fight for nationwide legalization. However, it is most definitely a step in the right direction for the world of marijuana and cannabis-based research.



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