The Food and Drug Administration (FDA) has accepted for Priority Review the supplemental New Drug Application (sNDA) for Epidiolex® (cannabidiol; Greenwich Biosciences) for the treatment of seizures associated with tuberous sclerosis complex (TSC), a rare genetic disease that causes tumor growth in vital organs of the body.

Epidiolex 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 precise mechanism by which cannabidiol exerts its anticonvulsant effects is unknown; it does not appear to exert its effects through interaction with cannabinoid receptors.

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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“More than 60% of individuals with TSC do not achieve seizure control with standard antiepileptic drug treatments,” said Justin Gover, GW’s Chief Executive Officer. “Today’s news is therefore important for TSC patients and their clinicians, and we look forward to working with the FDA during the review process to expand the Epidiolex product label for use in TSC.”

The FDA previously granted Orphan Drug designation to Epidiolex for the treatment of seizures associated with TCS. A Prescription Drug User Fee Act (PDUFA) target date of July 31, 2020 has been set for this application.

Epidiolex is already approved for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS) or Dravet syndrome in patients aged ≥2 years. 

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