GW Pharma starts Phase III trials for Tuberous Sclerosis Complex drug

pharmafile | April 11, 2016 | News story | Research and Development Epidiolex, GW Pharmaceuticals, Tuberous Sclerosis Complex, genetics, rare disease 

GW Pharmaceuticals Plc (Nasdaq: GWPH) said it has started late-stage trials for its Epidiolex (cannabidiol or CBD) to treat seizures in rare genetic disorder. 

Epilepsy is the most common symptom of the seizures associated with Tuberous Sclerosis Complex (TSC), the company said in a statement. 

The drug candidate is being studied in three indications including Dravet syndrome and Lennox-Gastaut syndrome, both rare forms of childhood-onset epilepsy. 

Chief Executive Justin Gover, says: “Our decision to evaluate Epidiolex in TSC is based on findings from the physician-led Epidiolex expanded access program, which found that TSC patients reported reductions in seizure activity. With the commencement of this trial, GW’s Epidiolex development program now encompasses three rare epilepsy indications with pivotal trials fully underway.” 

On March 14, GW announced positive results from the first of these pivotal trials in Dravet syndrome. 

Epidiolex, is a liquid formulation of pure plant-derived CBD, which is being developed for the treatment of a number of rare pediatric epilepsy disorders. 

Tuberous Sclerosis Complex (TSC) is a genetic disorder that causes non-malignant tumors to form in many different organs, with the brain and skin being the most commonly affected tissues. There are about 50,000 people in the US and nearly one million people worldwide estimated to have TSC. 

Anjali Shukla

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