FDA Approves Groundbreaking Drug for Treating Rare, Severe Forms of Epilepsy

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Officials with the FDA have approved cannabidiol oral solution (Epidiolex) for the treatment of seizures associated with 2 rare and severe forms of epilepsy, Lennox-Gastaut syndrome, and Dravet syndrome, in patients aged 2 years and older. This is the first FDA-approved drug that contains a purified drug substance derived from marijuana, and is the first FDA approval of a drug for the treatment of patients with Dravet syndrome.

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 (GW Research) is an oral solution indicated to treat seizures associated with Lennox-Gastaut syndrome (LGS), and Dravet syndrome in patients aged 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. This is the first FDA approval of a drug to treat Dravet syndrome.

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.”

For more information, see the the full article at SpecialtyPharmacyTimes.com.

For more resources pertaining to epilepsy, check out Pharmacy Times' sister site, NeurologyLive. The Clinical Focus condition page serves as a hub for news, articles, videos, and newly released data from the field's most attended conferences.

References

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.
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.