LONDON—The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Epidiolex, an investigational cannabidiol product from biopharmaceutical company GW Pharmaceuticals plc, which discovers, develops and commercializes novel therapeutics through its proprietary cannabinoid product platform. The compound is indicated for the treatment of Dravet syndrome, a rare and treatment-resistant form of childhood epilepsy. It is an oral liquid formulation of a highly purified extract of cannabidiol, a non-psychoactive molecule from the cannabis plant.
The use of cannabinoids in medicine has grown in popularity of late, with one of the leading indications being that of pain management. Past work has also shown that the compounds have use in the field of epilepsy, as cannabinoids can “modulate, neurotransmission, reduce neuro-inflammation and affect oxidative stress,” GW noted in a press release. In addition, certain cannabinoids also have a cumulative anti-convulsant effect “due to their ability to simultaneously modulate a number of endogenous systems to attenuate and/or prevent epileptic neuronal hyperexcitability.”
There are a number of channels linked to epileptogenesis, the process by which a normal brain becomes epileptic, and among them is ligand-gated ion channels, upon which plant cannabinoids can act, either in modulating transient receptor potential channels or in the agonism and antagonism of G-protein coupled receptors. And given that inflammation plays a role in epilepsy, the anti-inflammatory properties of some cannabinoids could prove an effective counter to the issue.
“GW is focused on advancing the Epidiolex development program as rapidly as possible with the aim of addressing the significant unmet need in children suffering from Dravet syndrome. The granting of Fast Track designation, in addition to Epidiolex having already received orphan drug designation from the FDA, represents significant additional support toward this objective,” Justin Gover, CEO of GW, said in a press release. “With GW having already opened an Investigational New Drug for Epidiolex, we are on track to commence a Phase 2/3 clinical trial in Dravet syndrome in the second half of this year.”
Dravet syndrome, which is also known as Severe Myoclonic Epilepsy of Infancy (SMEI) is a rare and catastrophic form of epilepsy in which seizures begin in the first year of life in infants that are otherwise developing normally. The initial seizures are generally prolonged events (status epilepticus), and in the second year of life, other types of seizures begin. All types demonstrate significant resistance to medical therapy, and the prognosis for Dravet syndrome patients is poor, particularly since they also face a higher incidence of sudden unexplained death in epilepsy and associated co-morbid conditions. Some 5,440 patients are afflicted with Dravet syndrome in the United States, with an estimated 6,710 patients in Europe, according to GW, though those numbers might be underestimated since Dravet syndrome is reportedly underdiagnosed.
This is not the only news for the Epidiolex program in recent months; in February, GW announced that the FDA had granted the drug candidate orphan drug status for the treatment of children with Lennox-Gastaut syndrome, a rare and severe form of childhood epilepsy. The company plans to conduct a clinical development program for the compound in Lennox-Gastaut as well. Epidiolex had previously been granted orphan drug designation for the treatment of Dravet syndrome in November 2013.