In a ground-breaking development, FDA in the year 2018 approved Epidiolex, a cannabidiol-based oral solution for the treatment of seizures. These two seizures are associated with two rare and severe forms of epilepsy — called Lennox-Gastaut Syndrome and Dravet Syndrome. Mostly, these seizures affect children aged two or more. Epidiolex, manufactured using purified drug substance derived from a marijuana plant, was the first such drug that got a green signal from the US department. The Epidiolex approval by the US FDA proved to be a big boost to CBD proponents. It also opened a new window of opportunities for thousands of entrepreneurs who believed in the power of medical marijuana.
What are CBD and Epidiolex?
For those of you who don’t know much about CBD, it’s one of the 500 compounds found in a cannabis Sativa or a hemp plant. CBD is the main compound that forms the basis of Epidiolex. This chemical, which is extracted from hemp using a solvent, has properties that give you a feel-good effect without causing ‘high’. It’s the other prominent compound called tetrahydrocannabinol or THC that holds primary psychoactive properties of marijuana. However, CBD is still a Schedule 1 substance under the US Controlled Substance Act, primarily because of its presence in cannabis or hemp plants.
Epidiolex is nothing but cannabidiol, which is extracted from cannabis or hemp plants. CBD has the powers to mimic natural endocannabinoids that act on brain receptors. These receptors can cause seizures if they are faulty. That’s where Epixiolex comes in. Researchers have found the lower intake of Epidiolex can have better safety and tolerability prospects.
What’s FDA take on Epidiolex approval?
The US FDA hailed the approval of cannabis-based Epidiolex as a reminder “that advancing sound development programs that properly evaluate active ingredients contained in marijuana can lead to important medical therapies”. With Epidiolex approval, the US FDA signalled that it’s committed to “careful scientific research and drug development”. While approving the drug, FDA Commissioner Scott Gottlieb, M.D. had said that it was possible to introduce cannabis-based health solutions into the mainstream by conducting “controlled clinical trials testing the safety and efficacy of a drug, along with careful review through the FDA’s approval process”.
Assuring about its safety, the FDA boss also said that because of the adequate and well-controlled clinical studies that supported this approval, prescribers can have confidence in the drug’s uniform strength and consistent delivery.
The FDA has also extended support to rigorous scientific research being carried out to explore potential medical uses of all marijuana-based products. At the same time, the FDA also said it is committed to taking action against those into selling CBD-based products with serious and unproven medical claims.
What are Dravet and Lennox Gastaut syndromes?
These two syndromes are rare forms of severe epilepsy among newborns and children. Dravet syndrome is a genetic condition. It usually appears during the first year of life. In this, a child feels fever-related seizures. As the condition deteriorates, new forms of seizures arise, including myoclonic seizures. Children may also suffer from status epilepticus, which is potentially a life-threatening health complication in which the body remains in the continuous state of “seizure activity”. Children suffering from this form of epilepsy usually have trouble developing language and motor skills. They also show hyperactivity and also fail to relate to others.
Lennox-Gastaut syndrome also affects young children with multiple seizures. Such patients usually face uncontrolled contraction in muscles, a condition also known as tonic seizures. Children suffering from this syndrome face difficulty in learning. Newborns can’t develop motor skills like sitting and crawling. The affected kids require regular assistance at home.
Both of these syndromes have a profound impact on the quality of life of patients. Experts say Epidiolex can treat the seizure problems arising out of Lennox-Gastaut syndrome. The drug has also been approved to treat Dravet syndrome patients too. The US Drug Enforcement Administration has also classified Epidiolex as a non-controlled substance, which means there are no federal curbs — that are usually imposed on controlled and Schedule 1 substance — on its access across the US markets.
Study on Epidiolex effectiveness
To assess the efficacy of Epidiolex, the FDA conducted three randomised clinical trials, which were double-blinded and placebo-controlled. The FDA had analysed the data on 516 patients who were suffering from both Lennox and Gastaut syndromes. When the results were analysed, it was found that those patients who consumed Epidiolex along with other treatment options experienced a fewer number of seizures than those given placebo.
Side-effects of Epidiolex
During the trials, the FDA also analyzed the side-effects of this CBD-based medication to ensure full-proof transparency. Some of the side-effects found during the study were sleepiness, sedation, and lethargy. Although, participants also experienced elevated liver enzymes, loss of hunger, diarrhea, fatigue, and weakness. Some participants also faced difficulty in sleeping. If you have been prescribed Epidiolex, make sure you obtain a medical guide along with the drug. This document contains all the information regarding the drug and the risks associated with it.
Bright future of Epidiolex
This year, the US FDA expanded the age for the Epidiolex use to 1 and older. Epidiolex has also been approved for tuberous sclerosis complex, a severe health complication in which benign tumours can cause epilepsy.
“This label expansion, including the expansion of the age range in all approved indications, further demonstrates that the FDA process can continue to enable broader patient access to appropriately tested regulatory approved cannabinoid medicines,” Justin Gover, CEO of UK-based GW Pharmaceuticals, the maker of Epidiolex said in an August press release.
The bottom line
Epidiolex has given high hopes to the cannabis industry in the US that’s otherwise seen with skepticism. The FDA has also made its intent clear that it wants to “work with product developers who are interested in bringing patients safe and effective, high-quality products”. The need of the hour, now, is to pursue authorities to allow in-depth research around cannabis products to gain maximum benefits from its magical medicinal properties.
