Epidiolex sNDA Seeking FDA Approval for Tuberous Sclerosis Complex Indication

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.”

References:

• GW Pharmaceuticals and Greenwich Biosciences Submit Supplemental New Drug Application to US FDA for Epidiolex (cannabidiol) for the Treatment of Tuberous Sclerosis Complex [news release]. GW Pharmaceuticals’ website. http://ir.gwpharm.com/news-releases/news-release-details/gw-pharmaceuticals-and-greenwich-biosciences-submit-supplemental. Accessed February 4, 2020.

• Trial Shows CBD Helpful for Tuberous Sclerosis Complex-Related Seizures, May Expand Treatment Options for Challenging Form of Epilepsy [news release]. https://www.aesnet.org/about_aes/press_releases/AES2019-CBD-TSC. Accessed February 4, 2020.