SK Biopharm announced that its innovative epilepsy drug cenobamate has shown efficacy in patients with generalized seizures in a Phase 3 clinical trial, marking a key step toward expanding its treatment indications.
The company (CEO Lee Dong-hoon) said on September 16 that it obtained positive topline results from a Phase 3 study evaluating cenobamate (U.S. brand name Xcopri) as an adjunctive therapy in adolescents and adults with generalized epilepsy.
Currently approved for the treatment of focal seizures in adults, cenobamate is now expected to broaden its indication to include adolescents and adults with primary generalized tonic-clonic (PGTC) seizures, the most widely recognized form of seizures among the general public.
The Phase 3 study was conducted across 122 trial sites in 12 countries, including the U.S. and South Korea, enrolling 169 patients aged 12 years and older with PGTC seizures. The trial assessed the efficacy and safety of cenobamate as adjunctive therapy compared with placebo.
Results showed a statistically significant reduction in seizure frequency: 71.9% in the cenobamate group versus 39.6% in the placebo group for the primary endpoint of PGTC seizure frequency reduction. Safety assessments indicated treatment-emergent adverse events (TEAEs) occurred in 60% of cenobamate-treated patients and 53% of those receiving placebo, with most events being mild to moderate in severity. The company said detailed findings will be presented at the 2025 American Epilepsy Society (AES) Annual Meeting in December.
PGTC seizures are known to carry a high risk of injury and sudden unexpected death in epilepsy (SUDEP). However, treatment options specifically approved for PGTC seizures remain limited, underscoring the significance of this study in addressing an unmet medical need. SK Biopharm said it plans to pursue a supplemental new drug application (sNDA) with the U.S. Food and Drug Administration (FDA) to add the PGTC indication.
“This outcome is especially meaningful as it allows us to bring new treatment opportunities to more patients,” said CEO Lee Dong-hoon. “The broad efficacy of cenobamate has now been clinically validated, and we expect this will further strengthen our foothold in the global antiepileptic drug market.”
