The FDA grants orphan drug designation to CDB liquid preparations for Epidiolex .. But what does that mean?
Epidiolex is a liquid formulation containing vegetable cannabidiol, or CBD. The drug was recently given orphan drug designation by the FDA. Here is all you need to know about this exciting development.
What is Epidiolex?
Epidiolex is a liquid formulation of cannabidiol of plant origin, or CBD .
The drug is made by a company called GW Pharmaceuticals. It is designed as a treatment for various pediatric epilepsy syndromes orphans.
The reason you’re hearing about Epidiolex in the news this week is due to FDA granted the drug called something orphan drug designation for the treatment of Dravet syndrome and two rare medical conditions characterized by dozens or even hundreds of seizures per day – Lennox-Gastaut syndrome.
The drug also received fast track designation by the FDA for Dravet syndrome, which means that in the future could be approved as a legitimate drug to treat the disease.
Currently, Epidiolex is an investigational drug that has not yet been approved for use by the FDA or any other national regulatory agency. However, this may change in the near future.
FDA grants greater access to Epidiolex for 420 children
first Epidiolex began headlines when the FDA granted 20 extended intermediate access (EA) investigational new drug (IND) to treat more than 420 children. I’m not doing that number, by the way. That’s the official number displayed on the website of GW Pharmaceuticals .
Later, a further 225 patients were FDA-approved 4 programs in the United States.
The vast majority of these IND (estimated at around 95%) are for patients between 1 and 17 years old.
When the FDA grants greater access to a particular drug without approval it means they are acknowledging that the drug is clearly effective treatment for a specific condition – but the organization does not yet know enough about the fully Apruébalo drug. future consideration is required. That’s the next step for Epidiolex.
The treatment of epilepsy
For years, we have seen evidence that cannabidiol reduces seizures in patients (especially children) with serious conditions -. Namely, Lennox-Gastaut and Dravet syndrome
In response, pharmaceutical companies looking to make a synthetic form of the CBD safely could give children and produced in the United States -. Cannabidiol, which currently occupies a legal gray area
How does Epidiolex work?
Epidiolex and cannabidiol are being intensively investigated at the moment in order to learn more about what that can be used to treat seizures.
The website of GW Pharmaceuticals goes into greater depth about why Epidiolex and cannabidiol can work:
“A series of laboratory experiments validated they have shown that certain cannabinoids can modulate neurotransmission [i], can reduce neuroinflammation [ii], and can affect oxidative stress [iii]. There is a cumulative anticonvulsant effect of certain cannabinoids due to its ability to simultaneously modulate a number of endogenous systems to mitigate and / or prevent epilepsy hyperexcitability neuronal [iv]. These include control of ion channel [v], inflammation [vi] and endogenous cannabinoid modulation [vii]. “
in simple terms, cannabidiol and Epidiolex can reduce inflammation and physical stress in the brain, which in turn leads to a lower likelihood of an attack.
However, GW Pharmaceuticals summarizes his explanation stating that “the exact mechanism of action of antiepileptic cannabinoids is a research topic in intensive course in GW Pharmaceuticals.” So we still have much work to do before we definitely know how Epidiolex works.
About GW Pharmaceuticals
GW Pharmaceuticals says its vision:
“is to be the world’s leading prescription cannabinoids, through the rapid profitable growth pharmaceutical products that meet unmet needs unclear. ”
To achieve this goal, GW Pharmaceuticals is developing a range of cannabinoid drugs and medicines. Epidiolex is one of the best known medicines of the company, although they are also known for Sativex. No drug has been approved by the FDA, although both are showing promise that could be approved in the future.
The company was founded in 1998 and listed on the NASDAQ Global Market (GWPH) and AIM (part of the London Stock Exchange).
GW Pharmaceuticals is based in Cambridge, UK, although the company has offices in Carlsbad, California and London, UK.