UCB has shared promising results from a late-stage study of fenfluramine in patients with an ultra-rare form of epilepsy.
The phase 3 trial has been evaluating adjunctive fenfluramine in 87 patients aged one to 35 years who have a CDKL5 deficiency disorder (CDD) diagnosis and uncontrolled seizures.
Occurring in approximately one in 40,000 to 60,000 live births, CDD is a developmental epileptic encephalopathy caused by variations in the CDKL5 gene. Symptoms typically appear at around six weeks of age, and the disorder causes severe neurodevelopmental delays that impact cognitive, motor, speech and visual function.
Fenfluramine, marketed under the brand name Fintepla, already holds approvals to treat seizures associated with Dravet syndrome and Lennox-Gastaut syndrome, two other rare and severe forms of epilepsy.
The study met its primary endpoint, based on the median percent change in countable motor seizure frequency between baseline and the titration plus maintenance phase compared to placebo.
UCB’s drug was also found to be well tolerated, and its safety profile was consistent with previous studies in Dravet and Lennox-Gastaut syndrome.
A long-term 52-week extension phase of the study is currently ongoing, with the aim of characterising the long-term safety and tolerability of fenfluramine in adult and paediatric CDD patients.
The company said it is planning to submit regulatory applications for fenfluramine in CDD to bring the drug to patients “as soon as possible”.
Fiona du Monceau, executive vice president, patient evidence at UCB, said the 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”.
“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,” du Monceau added.
The announcement comes less than three weeks after UCB unveiled positive new three-year data for its inflammatory disease drug Bimzelx (bimekizumab) in adults with active psoriatic arthritis, a condition characterised by skin plaques, swollen toes and fingers, and joint pain and stiffness.
