Epidiolex© is an investigational drug and has not been approved for use by the FDA or any other national regulatory agency
Orphan drug designation granted for Epidiolex in Dravet & Lennox-Gastaut syndromes by FDA, Fast Track designation granted by FDA for Dravet syndrome
FDA has granted 20 intermediate expanded access (EA) INDs to treat approximately 420 children
An additional 225 patients authorized by FDA under 4 U.S. State programs
The large majority of these INDs (estimated at 95%) are for patients > 1 year and < 17 years
Dr Geoffrey Guy being interviewed by CNN's Dr Sanjay Gupta
Orphan Childhood Epilepsy Program
Epilepsy is one of the most common neurological disorders in children. According to Russ in the February 2012 edition of Pediatrics, there is a point prevalence of 6.3 per 1000 children currently diagnosed with epilepsy or 466,000 childhood patients in the United States and 765,000 patients in Europe.
Specialists estimate that up to 20% of these cases show pharmacoresistance to current treatment (i.e., seizures that persist, despite accurate diagnosis and carefully monitored treatment with multiple antiepileptic drugs) and are deemed “medically intractable”. Furthermore it is recognized that many of those that do find relief often suffer side effects severe enough with their current medication that an alternative or adjunct is often sought.
In total, therefore, the size of the intractable pediatric epilepsy population is 93,200 patients in the United States and 153,000 in Europe.
Many cases of epilepsy are able to be classified and have clearly defined natural histories providing important information on the likelihood of seizure control and chance of remission. Some of the rarer electroclinical syndromes have very poor responses to treatment and negligible remission rates such as Ohtahara in neonates, Dravet in infants, Lennox-Gastaut in young children and progressive myoclonic epilepsies in adolescence. GW are investigating the potential of the cannabinoid CBD, purified and formulated as Epidiolex in children with these electroclinical syndromes. In response to the FDA's decision to allow "Investigational New Drug" studies with Epidiolex, quotes from GW's Chairman, Dr Geoffrey Guy, were recently reported in O'Shaughnessy's:
Our strategy for the development of Epidiolex in pediatric epilepsy is to initially concentrate on two orphan indication syndromes - Dravet Syndrome and Lennox-Gastaut Syndrome. We expect to further expand the market opportunity by either targeting additional orphan seizure disorders and/or by seeking approval for a wider indication of pediatric epilepsy refractory to current treatments.