In a first of its kind ruling, the U.S. Food and Drug Administration approved GW Pharmaceuticals’ Epidiolex, a cannabis-based treatment for seizures associated with two rare forms of epilepsy, Lennox-Gastaut syndrome (LGS) and Dravet syndrome.
In a first of its kind ruling, the U.S. Food and Drug Administration (FDA) approved GW Pharmaceuticals’ Epidiolex, a cannabis-based treatment for seizures associated with two rare forms of epilepsy, Lennox-Gastaut syndrome (LGS) and Dravet syndrome.
The approval of Epidiolex marks two firsts for the U.S. regulatory agency. The GW Pharma drug is the first FDA-approved drug that contains a purified drug substance derived from marijuana. Perhaps more importantly though for patients with Dravet syndrome, the approval is the first FDA treatment for patients with Dravet syndrome.
Cannabidiol (CBD) is a chemical component of the Cannabis sativa plant, more commonly known as marijuana. However, CBD does not cause intoxication. It’s the first in a new category of anti-epileptic drugs (AEDs), GW Pharma said.
“Today’s approval of Epidiolex is a historic milestone, offering patients and their families the first and only FDA-approved CBD medicine to treat two severe, childhood-onset epilepsies,” said Justin Gover, GW’s chief executive officer. “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.”
LGS and Dravet syndrome are rare and severe forms of epilepsy that are notoriously treatment-resistant. The diseases typically develop in early childhood. Because of the severity of the type of epilepsy, most patients with LGS and Dravet syndrome require multiple seizure medications and the majority are resistant to currently approved AEDs. The day-to-day impact of these conditions is significant with high rates of early mortality.
Epidiolex will be marketed in the U.S. by Greenwich Biosciences, the U.S. subsidiary of GW Pharmaceuticals plc. But before that can happen, the company must wait for the U.S. Drug Enforcement Administration to reschedule Epidiolex from its current Schedule I status before it can be made available to patients. Rescheduling is expected to occur within 90 days of the FDA approval, the company said. Access is expected to be similar to other branded AEDs and Epidiolex is expected to be available to appropriate patients by the fall of this year. The Europeans Medicine Agency is expected to rule on Epidiolex in the first quarter of 2019.
FDA Commissioner Scott Gottlieb noted that the approval of Epidiolex “serves as a reminder” that understanding and advancing development programs from the active ingredients in marijuana can lead to important medical therapies.
“Controlled clinical trials testing the safety and efficacy of a drug, along with careful review through the FDA’s drug approval process, is the most appropriate way to bring marijuana-derived treatments to patients. 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 in a statement.
The FDA approved Epidiolex following a Phase III trial that showed the marijuana-based treatment, taken along with other medications, was effective in reducing the frequency of seizures when compared with placebo in 516 patients with either Lennox-Gastaut syndrome or Dravet syndrome. The U.K.-based company will likely be looking for FDA approval of Epidiolex in other indications as well. GW has received Orphan Drug Designation from the FDA for the treatment of tuberous sclerosis complex (TSC) and infantile spasms (IS). GW is currently evaluating additional clinical development programs in other orphan seizure disorders including a Phase III trial in TSC.
GW Pharmaceuticals isn’t the only company working to develop cannabis-based treatments for disease. Other companies developing such treatments include Axim Biotechnologies, which is developing a cannabinoid-based chewing gum delivery method; KannaLife Sciences, which is researching oxidative and neuro-toxic stresses born from a variety of ailments and illnesses including Chronic Pain and Chronic Traumatic Encephalopathy; CURE Pharmaceuticals, which is developing cannabinoids for the treatment of a wide range of sleep disorders; as well as Columbia Care, which struck a licensing agreement with Australia-based nasal respiratory company Rhinomed for the collaborative development of nasally-delivered cannabis-based medicines.
Companies in the cannabis-based treatment space are hoping the FDA approval for Epidiolex will spur additional investors in their own treatments. Dean Petkanas, CEO of Kannalife Sciences, they “anticipate that investment capital may now be more likely to find its way into our end of the space,” due to the GW Pharma drug’s approval.
“We are encouraged by the market approval of Epidiolex as it affirms our judgment and decision to be a pharmaceutical company in what should be a burgeoning industry in the cannabinoid therapeutics market. And to this end, Wall Street may finally drop the old guard mantra that federal regulations prohibit investments in the space,” Petkanas said in a statement.
