GW Pharmaceuticals, a marijuana biopharma company based in the United Kingdom, is on track to submit its lead candidate Epidiolex to the FDA for approval during the first half of 2017 for the treatment of Dravet syndrome and Lennox–Gastaut syndrome (LGS), according to an article posted on the FierceBiotech website. Both indications are rare forms of childhood-onset epilepsy.
The drug is cannabidiol, one of many compounds in cannabis, but it does not have the psychoactive effects that are typically associated with marijuana. Epidiolex has received an orphan drug designation from the FDA for the treatment of patients with Dravet syndrome, LGS, tuberous sclerosis complex, or infantile spasms. It was also awarded a fast-track designation.
Cannabidiol has attracted attention as a potential treatment for seizures. Large-scale research on cannabinoids is difficult in the U.S., however, because of restrictions on the availability of marijuana for research use, since marijuana remains a highly limited, Schedule I drug, according to the U.S. Drug Enforcement Administration (DEA).
Epidiolex is an investigational drug and has not been approved for use by the FDA or any other national regulatory agency.
Epilepsy is one of the most common neurological disorders in children. Currently, 6.3 per 1,000 children (or 466,000 pediatric patients) are diagnosed with epilepsy in the United States each year. 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.” It has been estimated that 93,200 children have intractable pediatric epilepsy in the United States