A pediatric neurologist affiliated with RWJBarnabas was the co-lead investigator of the clinical trials.
The U.S. Food and Drug Administration on Monday approved the first cannabis-derived drug for sale, which has shown to control seizures in children with life-threatening forms of epilepsy.
Epidiolex, manufactured by GW Pharmaceuticals in the UK, is made from cannabidiol, the non-psychoactive part of the cannabis plant. It was made for children 2 and older who are diagnosed with Dravet syndrome and Lennox-Gastaut syndrome, two serious forms of epilepsy that cause life-long developmental delays.
It’s not clear how quickly the drug will be available for prescription.
The goal is have it available in pharmacies by the end of the year, said Orrin Devinsky, a pediatric neurologist and director of the New York University-Langone and Saint Barnabas Comprehensive Epilepsy Center in Livingston, who co-authored the research.
Marijuana liquid could be ally of kids with epilepsy, study says
First, the FDA must confer with the Drug Enforcement Administration to decide when to “reschedule” cannabidiol, better known as CBD, Devinsky said.
Cannabis, including CBD, is a “schedule 1” drug, meaning it has no medicinal value and poses a high risk for abuse. This status has stifled rigorous research, because the federal government will not pay for it.
Now with the approval of Epidiolex, a drug that will not produce an euphoric “high” feeling in the user — Devinsky said he expected CBD would be moved down to a schedule 4 or 5 drug, which is one that poses a low potential for abuse and dependence.
“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,” Devinsky said. “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 should provide a more reliable alternative than the homemade cannabis oil parents for years have made for the children, said Devinsky, who once treated Vivian Wilson of Scotch Plains, a toddler with Dravet syndrome.
Vivian’s parents, Brian and Meghan Wilson, elevated the shortcomings of the state’s medicinal marijuana program and helped pass a law that led to the availability of a wide range of strains. The family moved to Colorado in 2014 to take advantage of that state’s more advanced medicinal marijuana program.
Relying on homemade oil “is is a terrible system, creating products that likely have more variability and can potentially contribute to more side effects and less consistency,” Devinsky said.
Epidiolex is also the first prescription designed to treat children with Dravet syndrome, according to the FDA’s announcement.
Meghan Wilson, now a Denver resident, said she was “ecstatic” over the news.
“This is really monumental and in my opinion strongly correlated to the outcry from Dravet parents four to six years ago, begging neurologists for help accessing cannabis products for their children,” Wilson said.
“In the world of Dravet, there is always the fear of ‘running out of options’ and now, as a community, we have one more option which gives families more hope and peace of mind,” she added.
The FDA’s decision is historic but it is not unexpected.
An FDA advisory panel recently endorsed Epidiolex, saying any unknown risk associated with using the schedule 1 substance was worth it based on three clinical trials involving more than 500 children which showed children with life-threatening forms of epilepsy suffered fewer seizures while taking it.
In 2013, the FDA gave GW Pharmaceuticals of the United Kingdom permission to use the cannabidiol-derived drug, in clinical trials.
FDA Commissioner Scott Gottlieb said the agency “will continue to support rigorous scientific research on the potential medical uses of marijuana-derived products and work with product developers who are interested in bringing patients safe and effective, high quality products.”
“Because of the adequate and well-controlled clinical studies that supported this approval, prescribers can have confidence in the drug’s uniform strength and consistent delivery that support appropriate dosing needed for treating patients with these complex and serious epilepsy syndromes,” Gottlieb said.
Susan K. Livio may be reached at slivio@njadvancemedia.com. Follow her on Twitter @SusanKLivio. Find NJ.com Politics on Facebook.