NEW JERSEY: A nationally renowned pediatric neurologist at Saint Barnabas has gotten FDA approval to study whether a cannabis-based drug could prevent seizures in children diagnosed with severe forms of epilepsy.
The development is sure to be watched closely in New Jersey, where a number of families whose children have Dravet syndrome, a potentially deadly form of epilepsy, say they cannot obtain yet a useful form of medicinal marijuana through the state Department of Health.
The U.S. Food and Drug Administration gave Orrin Devinsky, director of the New York University and Saint Barnabas Comprehensive Epilepsy Center, Roberta Cilio of the University of California – San Francisco’s Neurology Department, and GW Pharmaceuticals of the United Kingdom permission to use the experimental drug, Epidiolex to treat 125 children with seizure disorders for whom traditional medicines have failed.Enrollment in the trial will begin next month, Devinsky said.
“I think this is a big step forward in the science of Cannabinoids,” said Devinsky, referring to the spectrum of active ingredients in marijuana. “We will finally get some data on epilepsy, and this should provide the basis to plan a randomized double-blind study.”
The drug is a liquid form of pure cannabidiol, a non-psychoactive component of the cannabis plant that according to anecdotal reports has shown to reduce the frequency and intensity of seizures in children. The FDA’s Nov. 15th approval also gives Epidiolex “orphan drug” status, providing GW full marketing rights to be the lone producer of the product for seven years, the company said in a statement.
Orphan diseases are rare, occurring in less than 200,000 of people in the nation. There are 5,440 documented cases of Dravet syndrome in the United States and 6,710 in Europe, although the actual number may be higher because the condition is under-diagnosed, according to a statement from the pharmaceutical company. The seizures can damage the brain, delay development and cause lifelong intellectual disabilities.