Epidiolex sNDA Seeking FDA Approval for Tuberous Sclerosis Complex Indication

February 4, 2020

A supplemental application for cannabidiol oral solution (Epidiolex, GW Pharmaceuticals and Greenwich Biosciences) has been submitted for the treatment of seizures associated with tuberous sclerosis complex.

GW Pharmaceuticals and Greenwich Biosciences Inc. have submitted a supplemental New Drug Application (sNDA) to the FDA for cannabidiol (Epidiolex) oral solution, CV for the treatment of seizures associated with tuberous sclerosis complex (TSC).1

The highly purified form of cannabidiol (CBD) is currently approved by the FDA to treat Lennox-Gastaut syndrome (LGS) or Dravet syndrome. If approved, this sNDA would extend its indication to include seizures associated with TSC, a rare genetic condition that is the leading cause of epilepsy.1

TSC primarily occurs in children and more than 60% of individuals with the disease do not achieve seizure control with standard treatments, according to GW.1

This sNDA submission is based on data from the phase 3 study evaluating the safety and efficacy of the CBD treatment in patients with TSC.2

The trial included 224 patients ages 1 to 65 years old with TSC who were randomized to receive either CBD of 25 mg/kg per day, CBD of 50 mg/kg per day, or a placebo (containing no CBD) for 16 weeks. All patients in the study had previously tried and discontinued a median of 4 antiepileptic drugs (AEDs) and were taking a median of 3 AEDs during the time of the study, such as valproate, vigabatrin, levetiracetam, or clobazam.2

Related: What Pharmacists Need to Know About Epidiolex

According to the results, the CBD reduced TSC-associated seizure frequency by approximately half: 49% for the 25 mg dose and 48% for the 50 mg dose compared with 27% for the placebo. Patients who received CBD experienced a greater reduction in seizures (36% for 25 mg and 40% for 50 mg) than with placebo (22%), according to the study. Additionally, caregivers reported overall improvement in 69% of those taking 25 mg of CBD, 62% of those taking 50 mg, and 40% of those who had the placebo.2

In terms of safety, adverse effects (AEs) were reported in 93% of patients in the 25 mg group, 100% in the 50 mg group, and 95% in the placebo group. The most common AEs included diarrhea, decreased appetite, and sleepiness. Treatment was discontinued due to AEs in 11% of the 25 mg group, 14% of the 50 mg group, and 3% in the placebo group. Additionally, 12% of the 25 mg patients and no placebo patients had elevated liver enzymes, and 81% who had that problem were also taking the AED valproate, according to the study.2

“The submission of this sNDA for Epidiolex is an important step towards the prospect of offering a new treatment option for those patients with TSC who battle difficult-to-treat seizures,” Justin Gover, CEO of GW Pharmaceuticals, said in a statement.1 “Having already obtained approval for Epidiolex in the treatment of seizures associated with Lennox-Gastaut syndrome and Dravet syndrome, this submission is based on positive phase 3 data showing that Epidiolex reduced TSC-associated seizures, which include both focal and generalized seizures types. We look forward to working with the FDA toward an expected approval later this year.”

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