FDA New Drug and Biologic Approvals, 2018 Midyear Review

Mary L Windle, PharmD


August 01, 2018

In This Article

Lennox-Gastaut Syndrome or Dravet Syndrome Seizures: Epidiolex (cannabidiol)

Indication: Indicated for seizures associated with Lennox-Gastaut syndrome (LGS) or Dravet syndrome (DS) in patients aged 2 years or older.

Mechanism: Cannabidiol is a structurally novel anticonvulsant, and the exact mechanism by which it produces anticonvulsant effects is unknown. It does not appear to exert its anticonvulsant effects through CB1 receptors, nor through voltage-gated sodium channels.


  • 2.5 mg/kg PO BID initially; after 1 week, dose may be increased to maintenance dose of 5 mg/kg BID.

  • If 5 mg/kg BID is tolerated and further seizure reduction is required, patient may benefit from a dosage increase up to a maximum recommended maintenance dosage of 10 mg/kg BID (ie, 20 mg/kg/day).

Approval was based on results from several studies that compared adding cannabidiol to conventional AEDs with placebo and the incidence of drop seizures from baseline. In one of the studies, 225 patients with LGS (mean patient age 15 years) were randomized to receive cannabidiol 20 mg/kg/day, 10 mg/kg/day, or placebo. The cannabidiol add-on doses were compared with placebo add-on over 14 weeks. During the 4-week baseline period, the median number of drop seizures was 85 in all groups combined. The median reduction from baseline in drop-seizure frequency per 28 days during the treatment period was 41.9% in the 20-mg cannabidiol group, 37.2% in the 10-mg group, and 17.2% in the placebo group. During treatment, 30 patients (39%) in the 20-mg group, 26 (36%) in the 10-mg group, and 11 (14%) in the placebo group had at least a 50% reduction from baseline in drop-seizure frequency. The odds ratio (OR) for 20 mg vs placebo was 3.85 (95% CI: 1.75-8.47; P<0.001) and the OR for 10 mg vs placebo was 3.27 (95% CI: 1.47-7.26; P = 0.003).



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