The U.S. Food and Drug Administration has approved Epidiolex to treat Dravet and Lennox-Gastaut syndromes, making history as the first therapy approved to treat Dravet, as well as the first marijuana-derived treatment to receive FDA approval for any indication.
GW Pharmaceutical‘s plant-derived cannabinoid medicine was approved to treat seizures associated with both Dravet and Lennox-Gastaut syndromes in patients ages 2 and older.
“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,” said Justin Gover, GW’s CEO, in a press release.
In April, following positive results from a Phase 3 trial, an FDA advisory committee voted unanimously to support Epidiolex’s approval. The therapy had previously been granted orphan status, fast-track designation, and priority review to support and speed up its approval in the U.S.
“In addition to another important treatment option for Lennox-Gastaut patients, this first-ever approval of a drug specifically for Dravet patients will provide a significant and needed improvement in the therapeutic approach to caring for people with this condition,” Billy Dunn, MD, director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research, said in an FDA press release.
Clinical development of Epidiolex included three randomized, controlled Phase 3 clinical trials and an open-label extension study in which Epidiolox added to other anti-epileptic therapies significantly reduced seizure frequency in Dravet and Lennox-Gastaut patients.
“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, MD, of NYU Langone Health’s Comprehensive Epilepsy Center and 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.”
Cannabidiol (CBD) is one of the most abundant compounds in the marijuana plant. Many clinical trials have been conducted or are currently underway testing CBD’s ability to treat a variety of ailments.
CBD is one of more than 80 compounds found in the marijuana plant, according to a statement from Scott Gottlieb, MD, FDA commissioner. Epidiolex is a purified form of the compound in a controlled dosage, which doctors can prescribe to their patients confident in the consistency of its formulation. CBD does not contain THC, the psychoactive ingredient in marijuana, so it cannot get patients high.
“This product approval demonstrates that advancing sound scientific research to investigate ingredients derived from marijuana can lead to important therapies. This new treatment provides new options for patients,” Gottlieb said in the statement.
“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. This is the approval of one specific CBD 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,” he said.
Because CBD is currently a Schedule 1 drug — those with a high potential for abuse and no medical value — GW cannot market Epidiolex until the Drug Enforcement Administration reclassifies it, which is expected to occur within 90 days.
Epidiolex is expected to be available to patients by this fall. The compound is also currently under review by the European Medicines Agency. A decision on whether to recommend approval should take place in the first quarter of 2019.
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