GW Pharmaceuticals anticipates filing a supplemental New Drug Application with the FDA for the new indication later this year.
GW Pharmaceuticals could be eying another approval for its cannabidiol epilepsy treatment Epidiolex after the company posted positive late-stage data in a study for the treatment of seizures associated with tuberous sclerosis complex (TSC), a rare and severe form of childhood-onset epilepsy.
On Monday, GW said Epidiolex hit the primary endpoint in the trial, which was the reduction in seizure frequency compared to baseline. The company, which is based in the U.K., but has a toehold in the U.S. through its subsidiary Greenwich Biosciences Inc., said results from both the 25 mg and 50 mg doses were similar. The two dosing levels provided seizure reductions of 48.6 percent and 47.5 percent from baseline respectively. That was in comparison to 26.5 percent for placebo, the company said. Additionally, GW said all secondary endpoints supported the primary endpoint and the safety profile of Epidiolex remained consistent.
GW Chief Executive Officer Justin Gover said the results from this trial represent the fifth positive Phase III results for Epidiolex. The company recently launched the drug for the treatment of seizures associated with Lennox-Gastaut syndrome and Dravet syndrome following the June 2018 approval of Epidiolex by the U.S. Food and Drug Administration. The approval of Epidiolex marked the first time the FDA approved a drug that contains a purified drug substance derived from marijuana and forced the reclassification of the medication by the Drug Enforcement Agency.
“These new data show EPIDIOLEX reduced TSC-associated seizures, which include both focal and generalized seizures types, expanding the body of reliable science supporting the use of Epidiolex. With these data, we look forward to submitting an sNDA to the FDA in the fourth quarter with the goal of expanding the product label in 2020 to help the lives of patients suffering with TSC,” Gover said in a statement.
TSC is a rare genetic condition that causes tumors, typically benign, to grow in many different organs of the body. The tumors can cause a number of health issues. Epilepsy is present in greater than 90 percent of patients with TSC and may progress to become intractable to medication, GW said. More than 60 percent of individuals with TSC and epilepsy do not achieve seizure control with standard treatments, the company added. TSC is a leading cause of genetic epilepsy, often occurring in the first year of life as either focal seizures or infantile spasms. Untreated early-onset seizures are associated with an increased risk of autism and intellectual disability.
The most common adverse events in patients receiving Epidiolex in this study include somnolence, decreased appetite, diarrhea, constipation, vomiting, transaminase elevations, pyrexia, seizure, cough and infections. Volker Knappertz, GW’s chief medical officer, noted that there was a lower incidence of known adverse events in the 25 mg group compared to the 50 mg group. When the company filed its supplemental New Drug Application, Knappertz said GW will seek approval for the lower dose.
