- UCB said it will present 18 abstracts at the 16th European Epilepsy Congress in Athens from Sept. 5 to Sept. 9.
- A post hoc analysis of a Phase 3 trial in CDKL5 deficiency disorder found fenfluramine was linked to a median 6.4 extra countable motor seizure-free days a month, versus 0.1 day with placebo, the company said.
- Fenfluramine is approved in the EU for Dravet syndrome and Lennox-Gastaut syndrome as add-on therapy for patients aged 2 years and older, UCB said, and is not approved for CDD or Rett syndrome by any regulator.
BRUSSELS, Belgium — UCB will present 18 research abstracts at the 16th European Epilepsy Congress in Athens from Sept. 5 to Sept. 9. The studies cover developmental and epileptic encephalopathies, Rett syndrome, acute seizure care, and the shift from pediatric to adult treatment.
The Brussels-listed biopharmaceutical company said in an emailed press release today that the program includes new analyses of fenfluramine, sold as Fintepla.
Donatello Crocetta, UCB’s chief medical officer, said the data “deepen our understanding of outcomes that matter most to individuals and families affected by developmental and epileptic encephalopathies (DEEs) and epileptic syndromes.” He also pointed to UCB’s recent acquisition of Neurona Therapeutics.
Analyses in Approved Syndromes
In adults with Lennox-Gastaut syndrome, a post hoc analysis of a randomized controlled trial (n=67) and its open-label extension (n=41) associated fenfluramine with improvements in caregiver-rated everyday executive functioning, UCB said.
Clinically meaningful improvement was defined as a Reliable Change Index of at least 90% on BRIEF-A T-scores. During the randomized trial, a greater share of adults on fenfluramine than on placebo showed that level of improvement on behavioral regulation (17% to 33% versus 12%), metacognition (33% versus 4%), and a global executive composite (28% to 38% versus 8%). The fenfluramine groups received 0.7 mg/kg/day (n=18) or 0.2 mg/kg/day (n=24), versus placebo (n=25).
In a prospective Dravet syndrome evaluation of 10 patients — five children aged 4 to 17 and five adults aged 24 to 46 — those treated for more than six months had generalized tonic-clonic seizure reductions of 25% and 100% in two pediatric patients (median 62.5%) and a median 74.5% reduction in three adults (range 69% to 80%), UCB said. Among the three patients who had reached one-year follow-up, the company said no statistically significant improvements in non-seizure outcomes were detected, though caregivers reported gains in motor function, alertness, and daily living skills.
CDD and Rett Studies
A post hoc analysis from a 14-week Phase 3 randomized, double-blind, placebo-controlled trial in CDKL5 deficiency disorder (n=86) found fenfluramine was associated with more countable motor seizure-free days than placebo, UCB said. Patients on fenfluramine had a median gain of 6.4 countable motor seizure-free days a month, versus 0.1 day with placebo. A higher share achieved more than seven such days a month (71.4% versus 31.8%).
Among fenfluramine-treated patients, those who gained at least seven countable motor seizure-free days a month were more likely to be rated by investigators as “much improved” or “very much improved” on the Clinical Global Impression-Improvement scale than non-responders (57.9% versus 21.7%). A separate post hoc time-to-event analysis found patients on fenfluramine took longer to return to baseline seizure counts than those on placebo (55 versus 29 days for countable motor seizures; 51 versus 29 days for all seizures).
UCB also presented the design of an ongoing Phase 3 randomized, double-blind, placebo-controlled study with an open-label extension (n=200) of fenfluramine in Rett syndrome. Coprimary endpoints are change from baseline to week 14 in the caregiver-completed Rett Syndrome Behaviour Questionnaire total score and an investigator-completed Clinical Global Impression of Change score. Fenfluramine is not approved for CDD or Rett syndrome by any regulatory authority, the company said.
Care Gaps and Rescue Treatment
Patient and caregiver studies in France, Italy, Poland, Spain, the UK, and the US found low awareness of the Rapid and Early Seizure Termination approach, at 15% (8 of 53 interview participants). Once the approach was explained, 67% (30 of 45 participants not currently using it) considered it feasible, UCB said. In a survey of 374 people, time to seizure cessation (32.4%) and mode of administration (26.6%) were the most important treatment attributes, and 88% (328 of 374) said they would be willing to wait 30 seconds to administer treatment.
A policy analysis of transition from pediatric to adult care for rare and complex epilepsies in the UK, France, Spain, Italy, Germany, and the United States found persistent gaps, including fragmented pathways and limited multidisciplinary support. About 50% of 57 respondents in an EpiCare reference-network survey reported having no written transition protocol, and only 12% provided dedicated transition coordinators and physical spaces, UCB said.
An anonymous internet survey of 582 caregivers found that those supporting people over 16 with developmental and epileptic encephalopathies (n=166) reported disruptive seizures, sleep problems, and/or behavior issues linked to temporary reductions in daily living and communication.
In the European Union, fenfluramine is approved as add-on therapy for seizures associated with Dravet syndrome and Lennox-Gastaut syndrome in patients 2 years of age and older.
UCB is headquartered in Brussels and listed on Euronext Brussels. It has more than 9,000 people in about 40 countries and reported 2025 revenue of €7.7 billion.