Nearly a year ago, UK drug manufacturer GW Pharmaceuticals filed its breakthrough epilepsy treatment for approval by U.S. regulators. The drug, Epidiolex, is derived from cannabidiol, or CBD, a non-psychoactive constituent of the cannabis plant. And on Tuesday, the FDA issued a report citing the “substantial evidence” GW Pharma had provided on the drug’s effectiveness. Could Epidiolex be the first cannabis-based drug to get FDA Approval?
Epidiolex: The Cannabis-Based Drug That Can Beat Epilepsy
There are many different types of epilepsy. But Dravet syndrome and Lennox-Gastaut syndrome are two of the severest forms of the disease. They’re also two of the hardest to treat.
Dravet and Lennox-Gastaut particularly impact children, who can suffer dozens and sometimes hundreds of seizures each month. Upwards of 20 percent of children with Dravet syndrome die before they reach adulthood.
The fact that CBD is non-psychoactive and doesn’t get users “high” makes it especially well-suited as a cannabis treatment for children. And GW Pharmaceuticals has formulated Epidiolex with this age group in mind.
Epidiolex is essentially a purified form of CBD. The CBD content comes from a strain of marijuana bred to produce high concentrations of cannabidiol. And in a double-blind, placebo-controlled study, the drug did remarkably well.
In March 2016, GW Pharma published the results of the first phase of its study. Researchers found that Epidiolex cut monthly seizures by 39 percent in children with Dravet syndrome.
But it wasn’t until May of last year that full results from the 120-patient study appeared in The New England Journal of Medicine, providing strong evidence that Epidiolex could significantly reduce seizure frequency and intensity in children with Dravet and Lennox-Gastaut syndromes.
Dr. Orrin Devinsky, director of the Comprehensive Epilepsy Center at New York University Langone Medical Center, said: “This study clearly establishes cannabidiol as an effective anti-seizure drug for this disorder and this age group.”
Confident in their successful results, GW Pharma filed Epidiolex for FDA approval shortly thereafter.
FDA Report Indicates Epidiolex’s Chances of Approval Are High
According to Bloomberg, GW Pharma is enjoying a bump in its share prices after an FDA report issued Tuesday indicated the agency is prepared to support the approval of the drug company’s experimental epilepsy treatment. If it does, Epidiolex will be the first cannabis-based drug to get FDA approval.
The FDA’s report did cite a few concerns, however. Among them was the issue of Epidiolex increasing the risk of liver injury. But the report also said that these side-effects could be managed.
“The risk-benefit profile established by the data in the application appears to support approval of cannabidiol,” FDA said in the report. Approval of Epidiolex could, therefore, pave the way for other CBD-based drugs.
“Epidiolex, if approved, will mark a sea change in the acceptability of cannabinoids as therapy,” said GW CEO Justin Gover. It’s also worth noting that FDA approval would mean patients’ insurance could cover the cost of the drug.
GW Pharma plans to market Epidiolex as a treatment for seizures caused by two forms of epilepsy, Lennox-Gastaut and Dravet syndromes, for children 2 years and older.
The company will sell the drug as an oral spray with a kid-friendly strawberry flavor.
The Final Hit: Could This Be The First Cannabis-Based Drug To Get FDA Approval?
Experts expect regulators to make a final decision on Epidiolex’s approval by June 27. On Thursday, officials with the FDA will meet with outside-agency advisors for additional input.
Even though the FDA has approved a handful of other drugs made from synthetic cannabinoids, GW Pharma’s Epidiolex would be the first cannabis-based drug to get FDA approval that actually comes from a marijuana plant.