GW Pharma’s cannabidiol (CBD) has been granted orphan drug designation by European regulators for the treatment of tuberous sclerosis (TS).
TS is a potentially life-threatening, genetic disease that causes benign tumors to grow in the brain and on other vital organs.
Up to 80 to 90 percent of individuals with TS will develop epilepsy during their lifetime, with onset typically in childhood, and up to two-thirds fail to respond adequately to available medical therapies.
GW is currently recruiting a Phase III clinical trial designed to investigate Epidiolex (cannabidiol) as an adjunctive therapy for the treatment of seizures associated with TS, with data expected in the second half of 2018.
If the data are favourable, GW expects to submit regulatory applications in 2019 for Epidiolex in TS in both the US and Europe, the firm noted.
GW has already submitted regulatory applications for Epidiolex on both sides of the Atlantic for the treatment of seizures associated with Lennox-Gastaut syndrome and Dravet syndrome.
“TS represents a near term opportunity to expand the potential indications for Epidiolex and reflects GW’s ongoing commitment to addressing the needs of patients with highly treatment-resistant seizures,” said the firm’s chief executive Justin Gover.
The drug already has orphan drug designation for the treatment of TS in the US.