Rare Daily Staff

The U.S. Food and Drug Administration approved GW Pharmaceuticals Epidiolex for the treatment of seizures associated with Lennox-Gastaut syndrome or Dravet syndrome. It is the first prescription pharmaceutical formulation of plant-derived cannabidiol and the first in a new category of anti-epileptic drugs.

LGS and Dravet syndrome, which develop in childhood, are rare, severe forms of epilepsy that are treatment-resistant. Most patients with LGS and Dravet syndrome require multiple seizure medications and the majority are resistant to currently approved anti-epileptic drugs. The day-to-day impact of these conditions is significant with high rates of early mortality.

Epidiolex is a pharmaceutical formulation of pure cannabidiol for the treatment of seizures associated with LGS or Dravet syndrome in patients two years of age or older. It does not produce the high associated with marijuana. GW has received Orphan Drug designation from the FDA for Epidiolex for the treatment of tuberous sclerosis complex and infantile spasms. The Company has also received Orphan Designation from the European Medicines Agency for Epidiolex for the treatment of LGS, Dravet syndrome, West syndrome and TSC. GW is currently evaluating additional clinical development programs in other orphan seizure disorders including a phase 3 trial in TSC.

“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 CEO.

“LGS and Dravet syndrome are two of the most severe and difficult-to-treat forms of childhood-onset epilepsy. These children and their families face a long and challenging road and very few achieve adequate seizure control,” said Elizabeth Thiele, director of the pediatric epilepsy program at Massachusetts General Hospital, professor of neurology at Harvard Medical School and a lead investigator in the Epidiolex clinical program. “Based on numerous clinical trials, this medication may help meet the need for this specific pediatric patient population and is now the first to be approved by the FDA in Dravet syndrome.”

The Epidiolex clinical development program included three randomized, controlled phase 3 clinical trials and an open-label extension study. In the Phase 3 studies, published in The New England Journal of Medicine and Lancet, Epidiolex added to other antiepileptic therapies significantly reduced the frequency of seizures in patients with LGS and Dravet syndrome.

The most common adverse reactions that occurred in Epidiolex-treated patients were somnolence; decreased appetite; diarrhea; transaminase elevations; fatigue, malaise, and asthenia; rash; insomnia, sleep disorder and poor quality sleep; and infections. The company’s development program represents the only well-controlled clinical evaluation of a cannabinoid medication for patients with LGS and Dravet syndrome.

“For those living with intractable seizures caused by LGS and Dravet syndrome, Epidiolex represents a true medical advancement,” said Philip Gattone, president and CEO of the Epilepsy Foundation. “Clinical development for these rare and severe conditions is essential, and today’s news brings hope for these patients and their families that a new treatment option may have the potential to help better control their seizures.”

Epidiolex will be marketed in the United States by Greenwich Biosciences, the U.S. subsidiary of GW Pharmaceuticals. As part of the approval process, the U.S. Drug Enforcement Agency must reschedule Epidiolex from its current status as a Schedule I as a marijuana derivative before it can be made available to patients. Rescheduling is expected to occur within 90 days. Access is expected to be similar to other branded anti-epileptic drug and Epidiolex is expected to be available to appropriate patients by Fall 2018.

Epidiolex is currently under review by the European Medicines Agency for the treatment of seizures associated with LGS and Dravet Syndrome. An EMA decision on whether to recommend approval is expected in the first quarter of 2019.

June 26, 2018

Photo: Justin Gover, CEO of GW Pharmaceuticals