GW Pharmaceuticals has submitted a supplemental New Drug Application (sNDA) to expand the indication of their cannabidiol oral solution, Epidiolex­® to treat seizures associated with tuberous sclerosis complex (TSC).

Epidiolex is currently approved in the US to treat seizures associated with Lennox-Gastaut syndrome and Dravet syndrome. It is a pharmaceutical formulation of highly purified cannabidiol (CBD); in 2018 it became the first approved drug to contain an active ingredient derived from marijuana as well as the first treatment approved for patients with Dravet syndrome.

The sNDA is supported by data from a phase 3 trial that included 224 patients with drug-resistant epilepsy associated with TSC. Patients were randomized to receive either Epidiolex at a dose of 25mg/kg/day (CBD25) or 50mg/kg/day (CBD50) or matched placebo for 16 weeks (4-week titration and 12-week maintenance phase). Results showed a significantly greater reduction from baseline in TSC-associated seizures with Epidiolex­ compared with placebo (49% with CBD25 vs 27% with placebo; P =.0009; 48% with CBD50 vs 26.5% with placebo; P=.0018).

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Tuberous sclerosis complex is a genetic disorder that causes tumors, which can lead to seizures, developmental delay, and autism. According to the TSC Alliance, there are ~50,000 individuals who have TCS in the US. 

The Food and Drug Administration previously granted Orphan Drug designation to Epidiolex­ for the treatment of TSC. Orphan designation is granted to treatments intended for diseases and conditions that affect fewer than 200,000 individuals in the US. 

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