GW Pharmaceuticals, along with U.S. subsidiary Greenwich Biosciences, has submitted a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for Epidiolex® (cannabidiol) oral solution, CV. The sNDA seeks to expand the Epidiolex label to include the treatment of seizures associated with Tuberous Sclerosis Complex (TSC), a rare genetic condition. Epidiolex is currently approved in the U.S. to treat seizures associated with Lennox-Gastaut syndrome (LGS) or Dravet syndrome and has been granted Orphan Drug Designation from the FDA for the treatment of TSC.
"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," said CEO, Justin Gover. "Having already obtained approval for Epdiolex 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."
TSC is a condition that causes mostly benign tumors to grow in vital organs of the body including the brain, skin, heart, eyes, kidneys and lungs and is a leading cause of genetic epilepsy. TSC is typically diagnosed in childhood. More than 60% of individuals with TSC do not achieve seizure control4 with standard treatments.
Subscribe to our e-Newsletters
Stay up to date with the latest news, articles, and events. Plus, get special offers
from American Pharmaceutical Review – all delivered right to your inbox! Sign up now!
The sNDA is supported by data from a Phase 3 safety and efficacy study, results of which were recently presented at the American Epilepsy Society 2019 annual meeting. The study met its primary endpoint with patients treated with Epidiolex 25 mg/kg/day experiencing a significantly greater reduction from baseline in TSC-associated seizures compared to placebo (49% vs 27%; p=0.0009). Results for the 50 mg/kg/day dose group were similar, with seizure reductions of 48% from baseline vs 26.5% for placebo (p=0.0018). All key secondary endpoints were supportive of the effects on the primary endpoint. The safety profile observed was consistent with findings from previous studies, with no new safety risks identified.
Tuberous sclerosis complex (TSC) is a rare genetic condition that affects approximately 40-80 thousand individuals in the U.S. and nearly one million people worldwide. At least two children born each day will develop TSC, with an estimated prevalence of one in 6,000 newborns. The condition causes mostly benign tumors to grow in vital organs of the body including the brain, skin, heart, eyes, kidneys and lungs and is a leading cause of genetic epilepsy. TSC often occurs in the first year of life with patients suffering from either focal seizures or infantile spasms and is associated with an increased risk of autism and intellectual disability. The severity of the condition can vary widely. In some children the disease is very mild, while others may experience life-threatening complications.
Seizures are present in about 85% of patients with TSC. More than 60% of individuals with TSC do not achieve seizure control with standard treatments such as antiepileptic drugs, epilepsy surgery, ketogenic diet, or vagus nerve stimulation compared to 30-40% of individuals with epilepsy who do not have TSC who are drug resistant.