GW Pharmaceuticals selects infantile spasms for Epidiolex development program

The company secured orphan drug designation from the US Food and Drug Administration for Epidiolex (cannabidiol or CBD) to treat IS.

A two-part pivotal Phase 3 study is expected to be started in the fourth quarter of this year.

Epidiolex is a liquid formulation of pure plant-derived cannabidiol (CBD), which is in development to treat several rare pediatric epilepsy disorders.

The drug is currently under development to treat Dravet syndrome, Lennox-Gastaut syndrome and Tuberous Sclerosis Complex.

GW Pharmaceuticals CEO Justin Gover said: "We are pleased to add infantile spasms as a fourth target indication for Epidiolex, demonstrating GW’s ongoing commitment to addressing the significant unmet medical need within the field of pediatric epilepsy.

"Currently, there are limited treatment options for children suffering from infantile spasms and outcomes for patients with the disorder include higher mortality, ongoing development of additional seizure disorders as the patient matures, and often severe cognitive and developmental delay."

The company carried out pre-clinical research of CBD in epilepsy since 2007. It demonstrated significant anti-epileptiform and anticonvulsant activity using several in vitro and in vivo models and reduced seizures in acute animal models of epilepsy with lesser side effects than comparator anti-epileptic drugs.

GW is currently assessing further clinical development programs in other orphan seizure disorders.