UCB reports fenfluramine success in CDKL5 deficiency disorder

Belgian pharma UCB (Euronext: UCB) today announced that the Phase III study investigating the safety and efficacy of adjunctive fenfluramine in CDKL5 deficiency disorder (CDD) met its primary and key secondary endpoints.

The study is a randomized, double-blind, placebo-controlled, fixed-dose, multicenter study examining the efficacy, safety, and pharmacokinetics of adjunctive fenfluramine treatment in 87 children and adults aged 1 – 35, with a CDD diagnosis and uncontrolled seizures.

"These results pave the way for creating significant therapeutic progress and represent an important milestone in UCB’s mission to bring meaningful innovation to individuals and families affected by developmental and epileptic encephalopathies (DEEs). We are grateful to the patients, families, and researchers who made this progress possible, and we look forward to working with the health authorities to make treatment available as soon as possible,” said Fiona du Monceau, executive vice president, patient evidence, UCB.