GW Pharmaceuticals, a biopharmaceutical company focused on novel therapeutics, has announced the US Food and Drug Administration (FDA) has approved EPIDIOLEX (cannabidiol) oral solution, for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS) or Dravet syndrome in patients 2 years of age or older.
EPIDIOLEX is the first prescription pharmaceutical formulation of highly-purified, plant-derived cannabidiol (CBD), a cannabinoid lacking the high associated with marijuana and the first in a new category of anti-epileptic drugs (AEDs). Product availability is pending rescheduling, which is expected to occur within 90 days.
“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.
“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, which develop in childhood, are rare, severe forms of epilepsy that are notoriously treatment-resistant. 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.
“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,” said Dr Orrin Devinsky of NYU Langone Health’s Comprehensive Epilepsy Center and a lead investigator in the EPIDIOLEX clinical programme.
“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.”
“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 Dr Elizabeth Thiele, Director of the pediatric epilepsy programme at Massachusetts General Hospital, Professor of neurology at Harvard Medical School and a lead investigator in the EPIDIOLEX clinical programme.
“Based on numerous clinical trials, this medication may help meet the need for this specific paediatric patient population and is now the first to be approved by the FDA in Dravet syndrome.”
The EPIDIOLEX clinical development programme included three randomided, controlled Phase III clinical trials and an open-label extension study.
In the Phase III 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 programme 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 US by Greenwich Biosciences, the US subsidiary of GW Pharmaceuticals. As part of the approval process, EPIDIOLEX must be rescheduled from its current Schedule I 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 AEDs and EPIDIOLEX is expected to be available to appropriate patients by Fall 2018.
Outside the US, this medicine is currently under review by the European Medicines Agency (EMA) 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.
