Officials with the FDA have approved a novel cannabidiol (CBD) treatment for 2 rare and severe forms of epilepsy, making it the first FDA-approved medication that contains a purified drug substance derived from marijuana, according to a press release.¹

Epidiolex is an oral solution indicated to treat seizures associated with Lennox-Gastaut syndrome (LGS) and Dravet syndrome in patients 2 years and older. The first in a new category of antiepileptic drugs (AEDs), Epidiolex is a prescription formulation of highly-purified, plant-derived CBD, a cannabinoid lacking the high associated with marijuana.

LGS and Dravet syndrome are rare, severe forms of epilepsy that both typically develop in childhood and are associated with high rates of early morbidity. Most patients with LGS and Dravet syndrome experience frequent seizures and are resistant to currently approved AEDs, requiring treatment with multiple seizure medications. This is the first FDA approval of a drug to treat Dravet syndrome.

“The difficult-to-control seizures that patients with Dravet syndrome and Lennox-Gastaut syndrome experience have a profound impact on these patients’ quality of life,” Billy Dunn, MD, director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research, said in a FDA press release.¹ “In addition to another important treatment option for Lennox-Gastaut patients, this first-ever approval of a drug specifically for Dravet patients will provide significant and needed improvement in the therapeutic approach to caring for people with this condition.”

The approval is based on efficacy data from 3 clinical trials involving 516 patients with either LGS or Dravet syndrome. In the trials, Epidiolex, taken along with other antiepileptic medications, reduced the frequency of seizures compared with a placebo. The most common adverse reactions were somnolence, decreased appetite, diarrhea, transaminase elevations, fatigue, malaise, rash, insomnia, sleep disorder and poor quality of sleep, and infections.

“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,” Orrin Devinsky, MD, of NYU Langone Health’s Comprehensive Epilepsy Center and a lead investigator in the epidiolex clinical program, said in a GW Pharmaceuticals press release.² “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.”

According to GW, Epidiolex has also been granted orphan drug designation from the FDA for the treatment of tuberous sclerosis complex and infantile spasms. As part of the approval process, Epidiolex must be rescheduled from its current Schedule 1 before it can be made available to patients, which is expected to occur within 90 days.²

Further epilepsy coverage can be found on Specialty Pharmacy Times' new sister site, NeurologyLive.

References

1. FDA approves first drug comprised of an active ingredient derived from marijuana to treat rare, severe forms of epilepsy [news release]. FDA’s website. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm611046.htm?utm_campaign=06252018_PR_FDA%20approves%20marijuana-derived%20drug%20to%20treat%20two%20forms%20of%20epilepsy&utm_medium=email&utm_source=Eloqua. Accessed June 25, 2018.

2.

Biosciences Announce FDA Approval of EPIDIOLEX® (cannabidiol) oral solution — the First Plant-derived Cannabinoid Prescription Medicine [news release]. GW Pharmaceuticals’ website. http://ir.gwpharm.com/news-releases/news-release-details/gw-pharmaceuticals-plc-and-its-us-subsidiary-greenwich. Accessed June 25, 2018.