UCB Reports Promising Phase 3 Results for Fenfluramine in CDKL5 Deficiency Disorder and Future Plans
UCB Reports Promising Phase 3 Results for Fenfluramine in CDKL5 Deficiency Disorder
UCB, a prominent global biopharmaceutical company, has revealed positive results from the Phase 3 study investigating the efficacy and safety of its drug, fenfluramine, in treating CDKL5 deficiency disorder (CDD). This clinical trial has successfully met its primary and several key secondary endpoints, marking a significant step in UCB's ongoing commitment to tackle developmental and epileptic encephalopathies (DEEs).
Background on CDKL5 Deficiency Disorder
CDKL5 deficiency disorder is a rare but severe form of epilepsy that presents in early infancy, often characterized by frequent seizures, profound developmental delays, and other neurological impairments. Approximately 1 in every 40,000 to 60,000 live births are affected, with initial symptoms typically appearing around six weeks of age. The impact on quality of life and the significant unmet medical need in this population highlight the importance of new treatment options.
Details of the Phase 3 Study
The Phase 3 study was a randomized, double-blind, placebo-controlled trial assessing the effects of adjunctive fenfluramine in 87 patients aged between one and 35 years diagnosed with CDD and experiencing uncontrolled seizures. The primary efficacy endpoint measured the median percentage change in countable motor seizure frequency (CMSF) from baseline, comparing the outcomes to a placebo group. The study's results are expected to be detailed in an upcoming scientific meeting, showcasing UCB's innovative approach to developing effective treatments for this challenging condition.
Fiona du Monceau, UCB's Executive Vice President of Patient Evidence, expressed gratitude towards the patients, their families, and the researchers involved in the trial. She emphasized that these results represent a pivotal milestone in their mission to provide significant therapeutic advancements for individuals with DEEs.
Commitment to Innovation
UCB aims to submit for regulatory approval to provide this promising treatment option to those living with CDD as soon as feasible. Fenfluramine has been previously authorized in the U.S. for treating seizures associated with Dravet syndrome and Lennox-Gastaut syndrome, but it has not yet received approval for CDD. The ongoing open-label long-term extension phase of the study will further evaluate its safety and tolerability over a 54-week period.
The encouraging results underscore UCB's commitment to driving scientific innovation and addressing pressing healthcare needs. As they work toward making treatment accessible for CDD, they continue to explore ways to improve the quality of life for individuals affected by severe neurological disorders.
Conclusion
The positive outcome of this Phase 3 study signifies a beacon of hope for the CDD community, demonstrating UCB's dedication to pioneering research and the pursuit of innovative solutions in the realm of rare and complex disorders. The company remains focused on enhancing treatment options and fulfilling the unmet needs in the realm of developmental and epileptic encephalopathies. For follow-up inquiries, UCB has provided contact information for their communications and investor relations teams.