GW Pharmaceuticals’ cannabis drug gets FDA orphan drug status

The orphan drug status to the drug, Epidiolex, entitles GW Pharmaceuticals to a seven-year exclusive market period.

TSC is the third orphan indication that the company is targeting within its Epidiolex clinical development program.

The program features four Phase 3 pivotal trials in Dravet syndrome and Lennox-Gastaut syndrome, both rare and catastrophic forms of childhood-onset epilepsy.

Earlier this month, GW started a phase 3 clinical trial of Epidiolex as an adjunctive therapy to treat seizures associated with TSC.

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

The pre-clinical research of CBD in epilepsy, undertaken by GW Pharmaceuticals since 2007, demonstrated that CBD has significant anti-epileptiform and anticonvulsant activity using various in vitro and in vivo models.

It also has the ability to treat seizures in acute animal models of epilepsy with fewer side effects , compared to existing anti-epileptic drugs.

The company has so far secured orphan drug designation from the FDA for Epidiolex to treat Dravet syndrome, Lennox-Gastaut syndrome and TSC.

GW has also received fast track designation from the FDA and orphan designation from the European Medicines Agency for Epidiolex for the treatment of Dravet syndrome.

The company is currently evaluating further clinical development programs in other orphan seizure disorders.