Epidiolex Review – Should You Try It?
FDA Grants Orphan Drug Designation to CBD Liquid Formula Epidiolex.. But what does that mean?
Epidiolex is a liquid formula containing plant-derived cannabidiol, or CBD. The drug was recently given orphan drug designation by the FDA. Here’s everything you need to know about this exciting development.
What is Epidiolex?
Epidiolex is a liquid formulation of plant-derived Cannabidiol, or CBD.
The drug is made by a company named GW Pharmaceuticals. It’s designed as a treatment for various orphan pediatric epilepsy syndromes.
The reason you’re hearing about Epidiolex in the news this week is because the FDA granted the drug something called Orphan Drug Designation in the treatment of Dravet syndrome and Lennox-Gastaut syndrome – two rare medical conditions characterized by dozens or even hundreds of seizures per day.
The drug also received fast track designation by the FDA for Dravet syndrome, which means it could be approved in the future as a legitimate medicine for the treatment of the condition.
Currently, Epidiolex is an investigational drug that has not yet been approved for use by the FDA or any other national regulatory agency. However, that may change in the near future.
The FDA Grants Expanded Access to Epidiolex for 420 Children
Epidiolex first started making headlines when the FDA granted 20 intermediate expanded access (EA) Investigational New Drugs (INDs) to treat approximately 420 children. I’m not making that number up, by the way. That’s the official number listed on the GW Pharmaceuticals website.
Later on, an additional 225 patients were authorized by the FDA under 4 US State programs.
The large majority of these INDs (estimated at around 95%) are for patients between 1 and 17 years of age.
When the FDA grants expanded access to a certain medicine without approving it, it means they’re acknowledging that the drug is clearly an effective treatment for a specific condition – but the organization doesn’t yet know enough about the drug to fully approve it. Future testing is required. That’s the next step for Epidiolex.
Treatment for Epilepsy
For years, we’ve seen evidence that cannabidiol reduces epileptic seizures in patients (particularly children) with serious conditions – namely Lennox-Gastaut syndrome and Dravet syndrome.
In response, pharmaceutical companies sought to make a synthetic form of CBD that could safely be given to children and produced in the United States – where cannabidiol currently occupies a legal grey area.
How Does Epidiolex Work?
Epidiolex and cannabidiol are being intensely researched as we speak in order to learn more about why they can be used to treat seizures.
The GW Pharmaceuticals website goes into greater depth about why Epidiolex and cannabidiol may work:
“A series of validated laboratory experiments have shown that certain cannabinoids can modulate neurotransmission[i], can reduce neuro-inflammation[ii], and can affect oxidative stress[iii]. There is a cumulative anti-convulsant effect from certain cannabinoids due to their ability to simultaneously modulate a number of endogenous systems to attenuate and/or prevent epileptic neuronal hyperexcitability[iv]. These include ion channel control[v], inflammation[vi] and endocannabinoid system modulation[vii].”
In layman’s terms, cannabidiol and Epidiolex can reduce inflammation and physical stress in the brain, which then leads to a reduced likelihood of experiencing a seizure.
Nevertheless, GW Pharmaceuticals sums up their explanation by stating that “the precise mechanism of action of the anti-epileptic cannabinoids is a subject of intensive ongoing research at GW Pharmaceuticals.” So we still have more work to do before we definitively know how Epidiolex works.
About GW Pharmaceuticals
GW Pharmaceuticals claims that its vision:
“is to be the global leaders in prescription cannabinoid medicines, through the rapid cost-effective development of pharmaceutical products which address clear unmet needs.”
To achieve that goal, GW Pharmaceuticals is developing a range of cannabinoid drugs and medicines. Epidiolex is one of the company’s best-known drugs, although they’re also known for Sativex. Neither drug has yet been approved by the FDA, although both are showing promise that they could be approved in the future.
The company was founded in 1998 and is listed on both the NASDAQ Global Market (GWPH) and AIM (part of the London Stock Exchange).
GW Pharmaceuticals is headquartered in Cambridge, United Kingdom, although the company maintains offices in Carlsbad, California and London, UK.
