Positive results from GEMZ Phase III in CDKL5 deficiency disorder

Belgium’s UCB (Euronext: UCB) has presented positive efficacy and safety results from the GEMZ Phase III study investigating adjunctive Fintepla (fenfluramine) in children and adults with CDKL5 deficiency disorder (CDD) at the American Epilepsy Society (AES) meeting, Atlanta, USA.

The trial met its primary endpoint and key secondary endpoints, demonstrating a statistically significant reduction in countable motor seizure frequency (CMSF) and a clinically meaningful improvement on the Clinical Global Impression-Improvement (CGI-I) scale, compared with placebo.

“UCB is proud to share these important results with the medical community at AES, especially given the significant unmet need in CDD. Families affected by this ultra-rare condition face immense daily challenges with frequent, treatment-resistant seizures that are profoundly disruptive to daily life. These trial results emphasize the impact that seizure control can have on the lives of patients and their families, and we look forward to working with health authorities to make this treatment available as soon as possible,” said Fiona du Monceau, executive vice president, patient evidence, UCB.