A U.S. Food and Drug Administration advisory committee on April 19 recommended approval of the first cannabidiol (CBD) medicine for prescription use in two severe pediatric epilepsy syndromes. The vote was 13-0.
Jose E. Cavazos, M.D., Ph.D., professor of neurology and physiology at UT Health San Antonio and assistant dean of the institution’s M.D./Ph.D. program, was invited to review clinical studies of the drug, which would be sold under the brand name Epidiolex.
“I was asked to evaluate the scientific credibility of the evidence—whether there was sufficient research data—to recommend approval of this drug to treat Lennox-Gastaut and Dravet syndromes, which are two types of catastrophic epilepsy,” Dr. Cavazos said.
Children with these syndromes experience many seizures a day and treatment choices are extremely limited.
CBD is derived from the Cannabis sativa plant, famous as the source of marijuana. CBD is a non-addictive component of the plant, in contrast to tetrahydrocannabinol (THC), which is a component that produces the high of marijuana and is addictive.
Epidiolex is a proprietary oral solution of plant-derived CBD that contains little (less than 0.1 percent) THC, and it does not give the euphoric or abuse potential effects of THC. Clinical trials indicate the medication can decrease the frequency of “drop attacks” and other symptoms of Lennox-Gastaut. In these attacks, a person suddenly stiffens and falls with the head facing forward. Children with Lennox-Gastaut may wear helmets to prevent head injuries.
Controlled CBD use has been legal in Texas since 2015 when lawmakers passed the Texas Compassionate Use Act. Earlier this year, CBD oil dispensaries were created, allowing families of children with epilepsy to obtain the oil with physician authorization. “The pharmaceutical-grade CBD is being dosed at a much higher dose (between 10 and 100-fold) than the most common dose of the CBD oil sold in Texas dispensaries,” Dr. Cavazos said.
Cannabidiol products are classified as Schedule 1 substances under the U.S. Controlled Substances Act. Schedule I substances are defined as drugs with no currently accepted medical use and a high potential for abuse. Dr. Cavazos indicated that the scientific data showed little or no potential of abuse for CBD in the double-blinded clinical trials presented to the FDA review panel.
Linda Leary, M.D., clinical associate professor of neurology and pediatrics in the Joe R. & Teresa Lozano Long School of Medicine at UT Health San Antonio, said FDA final approval, when granted, should result in the drug being rescheduled for use in the two childhood syndromes of catastrophic epilepsy.
Some Texas institutions, including UT Health San Antonio, are assisting families who want to try CBD in their children with difficult-to-control epilepsy, Dr. Leary said.
“One reason we are willing to do this is that at least we can help families to use it safely,” Dr. Leary said. “CBD is not viewed as a miracle, but is one more medication that could work to control seizures.”
The FDA decision is significant because it will affect insurance status and provide safety guidelines and dosing instructions, Dr. Leary said. No guidelines currently exist for use of the Texas dispensary products, leaving families and physicians little information.