The United States Food and Drug Administration approved a cannabis-based drug for the first time ever, according to the FDA.
A drug called Epidiolex was originally recommended for approval in April. The drug is used to treat epileptic syndromes in people 2 years and older. The syndromes treated include Dravet syndrom and Lennox-Gastaut syndrome. Both of those syndromes often involve seizures and signs of epilepsy in early childhood that span into adulthood. The FDA had until this week to make its final decision.
"This is an important medical advance. But it’s also important to note that this is not an approval of marijuana or all of its components," said FDA Commissioner Scott Gottlieb. "This is the approval of one specific CBD [cannobiodiol] medication for a specific use. And it was based on well-controlled clinical trials evaluating the use of this compound in the treatment of a specific condition."
Epidiolex is the first pharmaceutical form of cannabidiol (CBD). CBD is a cannabinoid that lacks the high often associated as a side-effect of recreational marijuana usage. The drug, created by UK-based company GW Phramaceuticals, is also the first of its kind in anti-epilepsy medications.
"Moreover, this is a purified form of CBD," Gottlieb continued. "It’s being delivered to patients in a reliable dosage form and through a reproducible route of delivery to ensure that patients derive the anticipated benefits. This is how sound medical science is advanced."
The GW Pharmaceuticals team said in a statement it was pleased to finally have such an important drug on the market to provide hope to two long and difficult types of childhood-onset epilepsy.
“Today’s approval of EPIDIOLEX is a historic milestone, offering patients and their families the first and only FDA-approved CBD medicine to treat two severe, childhood-onset epilepsies,” said Justin Gover, GW’s Chief Executive Officer. “This approval is the culmination of GW’s many years of partnership with patients, their families, and physicians in the epilepsy community to develop a much needed, novel medicine. These patients deserve and will soon have access to a cannabinoid medicine that has been thoroughly studied in clinical trials, manufactured to assure quality and consistency, and available by prescription under a physician’s care.”
Medical professionals noted they're finally able to prescribe their patients a solution they frequently get asked about during treatment.
“In my practice, I often see patients with these highly treatment-resistant epilepsies who have tried and failed existing therapies and are asking about CBD,” said Orrin Devinsky, M.D., of NYU Langone Health’s Comprehensive Epilepsy Center. Devinsky also served as a lead investigator in the EPIDIOLEX clinical program.
“I am delighted that my physician colleagues and I will now have the option of a prescription cannabidiol that has undergone the rigor of controlled trials and been approved by the FDA to treat both children and adults.”
The drug became a reality after extensive medical testing, Gottlieb noted.
"This research process – from early development through preclinical and clinical research – gives us a comprehensive understanding of a new drug," he said. "That includes an understanding of whether the new product is safe and effective for treating a particular medical condition, what the proper dosage is and for what populations it is safe and effective, how the new compound could interact with other drugs, or whether the new drug has side effects or other safety concerns."
Gottlieb explained this wouldn't be the only CBD-based medication hitting the market. However, more research could be needed into how CDB-based drugs affect other ailments before those drugs become approved by the FDA and other health organizations.
"Drugs derived from marijuana also are eligible for several programs that are intended to facilitate and expedite development and review of new drugs that address unmet medical needs in the treatment of serious or life-threatening conditions. Much of the work we’ve done to encourage research in this area has led to the approval action we took today."