The Food and Drug Administration (FDA) has approved Epidiolex (cannabidiol oral solution; GW Pharmaceuticals) for the treatment of seizures associated with Lennox-Gastaut syndrome and Dravet syndrome in patients ≥2 years of age. This marks the first approved drug that contains an active ingredient derived from marijuana as well as the first treatment approved for patients with Dravet syndrome.
The approval of Epidiolex was based on data from 3 randomized, double-blind, placebo-controlled trials (N=516) involving patients with either Lennox-Gastaut (Study 1 and 2) or Dravet syndrome (Study 3). The primary efficacy measure in Study 1 and 2 was the percent change from baseline in the frequency (per 28 days) of drop seizures over the 14-week treatment period. When taken with other medications, treatment with Epidiolex 20mg/kg/day resulted in a significantly greater median reduction in the frequency of drop seizures vs placebo (Study 1: -44% vs -22% [P=.01]; Study 2: -42% vs -17% [P<.01 for both]). In Study 3, the median reduction in the frequency of convulsive seizures was significantly greater in the Epidiolex 20mg/kg/day group vs placebo (-39% vs -13%; P<.01). For all studies, a reduction in seizures was seen within 4 weeks of treatment initiation and the effect remained generally consistent over the 14-week treatment period.
Sleepiness, sedation, lethargy, elevated liver enzymes, decreased appetite, diarrhea, rash, fatigue, malaise, weakness, insomnia, sleep disorder, poor quality sleep, and infections were among the most common adverse effects seen with Epidiolex treatment. The most serious risks include suicidal ideation and attempts, agitation, new or worsening depression, aggression, and panic attacks.
Epidiolex is currently classified as a Schedule I substance because it is a chemical component of the cannabis plant. Before it becomes available to patients, the treatment will need to be rescheduled; GW Pharmaceuticals expects this to occur within the next 90 days.
Epidiolex will be supplied as a 100mg/mL strawberry-flavored oral solution in 100mL bottles and is expected to be available for appropriate patients in Fall 2018.
For more information call (833) 424-6724 or visit GWPharm.com.