By Fineline Cube 
Belgium-based biopharma UCB (EBR: UCB) announced positive results from its Phase III GEMZ study, evaluating the safety and efficacy of fenfluramine as adjunctive therapy for CDKL5 deficiency disorder (CDD).
Drug Profile
Fenfluramine, a small-molecule antiseizure medication, was previously approved for Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS)-associated seizures. The GEMZ study investigated its use in CDD, a rare and severe developmental epileptic encephalopathy caused by pathogenic mutations in the CDKL5 gene on the X chromosome. CDD typically manifests in early infancy and presents high unmet medical needs with limited treatment options.
Clinical Trial Details
The GEMZ study enrolled 87 participants aged 1–35 years with confirmed CDD and uncontrolled seizures. The primary endpoint assessed the median percentage change from baseline in countable motor seizure frequency (CMSF) during titration and maintenance periods, comparing fenfluramine versus placebo.
Key Findings
The trial successfully met its primary endpoint and most key secondary endpoints. Fenfluramine demonstrated favorable tolerability and a safety profile consistent with previous reports.-Fineline Info & Tech