UCB announces positive results from GEMZ Phase 3 study of fenfluramine in CDKL5 deficiency disorder

The primary endpoint of the study is based on the median percent change in countable motor seizure frequency (CMSF) between baseline and the titration plus maintenance phase, comparing fenfluramine with the placebo group. Full results will be presented at an upcoming scientific meeting.

CDD is an ultra-rare DEE with refractory infantile-onset epilepsy and severe global neurodevelopmental delays resulting in intellectual, motor, cortical visual, and sleep impairments as major features. It is caused by pathogenic variants in the cyclin dependent kinase-like 5 (CDKL5) gene located on the X chromosome. It is estimated that CDD affects approximately 1 in 40,000 to 60,000 live births.

In the study, fenfluramine was generally well tolerated, and the safety profile was consistent with previous studies in DS/LGS.3 UCB is currently conducting an open-label, flexible-dose, long-term 54-week extension phase of the study to characterize the long-term safety and tolerability of fenfluramine in pediatric and adult individuals with CDD.

In the United States, fenfluramine oral solution is indicated for the treatment of seizures associated with Dravet syndrome and Lennox-Gastaut syndrome in patients 2 years of age and older. It is not approved for use in CDD by any regulatory authority worldwide.

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