US FDA backs GW treatment of rare child epilepsies
United States medical watchdog, the FDA, has approved a cannaboid-based solution from GW Pharmaceuticals in Cambridge and its US subsidiary Greenwich Biosciences to treat seizures that affect children suffering rare and severe forms of epilepsy.
EPIDIOLEX® is a treatment of seizures associated with Lennox-Gastaut syndrome (LGS) or Dravet syndrome in patients two years of age or older.
It 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.
Justin Gover, GW’s CEO said: “The 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.
“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.
Orrin Devinsky, MD, of NYU Langone Health’s Comprehensive Epilepsy Center and a lead investigator in the EPIDIOLEX clinical program said: “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.
“I am delighted that my physician colleagues and I will now have the option of a prescription cannabidiol that has undergone the rigour of controlled trials and been approved by the FDA to treat both children and adults.”
Elizabeth Thiele, MD, director of the paediatric epilepsy programme at Massachusetts General Hospital, professor of neurology at Harvard Medical School and another lead investigator in the EPIDIOLEX clinical programme added: “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. 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.”
EPIDIOLEX will be marketed in the US by Greenwich Biosciences. 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 autumn 2018. More information can be found at Epidiolex.com.
• PHOTOGRAPH SHOWS: Justin Gover