A new cannabis-based drug made by GW Pharmaceuticals has been approved by the U.S. Food and Drug Administration (FDA) for two separate trials involving children with epilepsy.
The drug is made from purified cannabidiol (CBD) – a non-psychoactive compound in marijuana – and is being marketed under the name Epidiolex, reports O’Shaughnessy’s.
So far, the FDA has approved two Investigational New Drug studies of Epidiolex for pediatric epilepsy, which are being led by Orrin Devinsky, MD, at the NYU School of Medicine, and Roberta Cilio, MD, PhD, at the University of California, San Francisco (UCSF). Each will involve 25 children with epilepsy, and other studies are awaiting approval.
If all goes as planned, GW Pharmaceuticals’ Chairman Geoffrey Guy, MD, expects more studies to begin within months.
“In the coming months, if the FDA is comfortable about how things are going, there will be a number of senior epileptologists in major university centers throughout the U.S., each treating a couple of dozen patients with various epilepsies.”
GW Pharmaceuticals is best known for its cannabis-based spray called Sativex, which is approved in over 20 countries for the treatment of multiple sclerosis symptoms.
However, the company has spent recent years developing a drug for epilepsy. Preclinical studies sponsored by the company show that CBD, along with a related cannabinoid called CBDV, have the potential to reduce epileptic seizures. Anecdotal reports also suggest that CBD-rich cannabis extracts could be effective in treating epilepsy in children.
Unlike Sativex, Epidiolex is a liquid medicine that can be administered with a syringe dropper. According to the company, the drug contains more than 98 percent CBD, along with trace amounts of other cannabinoids.
It is also THC-free, which will ensure that children are not getting high from the medicine.Dr. Guy says if early results are positive, the FDA could speed up the clinical trial processAccording to Dr. Guy, the FDA process came after parents of epileptic children began contacting the company in late 2012 hoping to obtain CBD. Since GW Pharmaceuticals was already working with the FDA on trials of Sativex, the company decided it made sense to seek research approval for Epidiolex.
Dr. Guy told O’Shaughnessy’s that he expects the trials to provide a better understanding of “what cannabidiol does in these different children groups, what benefit we can see, and how the results can best be measured.”
The clinical trial process will likely take a number of years to complete. However, Dr. Guy says if the drug shows promise in Phase I trials, the FDA could speed up the transition into Phase III.
Last month, GW Pharmaceuticals announced the start of clinical trials of the epilepsy drug in the UK.