UCB has announced new phase 3 clinical trial findings for Fintepla in patients with cyclin-dependent kinase-like-5 (CDKL5) deficiency disorder and stated that it intends to file for regulatory approval for this third epilepsy-related indication “as soon as possible.” The company shared the results during the American Epilepsy Society (AES) meeting in Atlanta.
The study enrolled 87 participants between the ages of 1 and 35 who were diagnosed with the ultra-rare CDKL5 disorder. According to UCB, the trial met its primary objective by demonstrating that Fintepla produced a statistically significant reduction in seizures compared with placebo. The company added that the study achieved two of its three secondary endpoints.
Fintepla received its first approval in 2020 for treating seizures linked to Dravet syndrome, a severe and uncommon form of epilepsy that affects an estimated 20,000 people in the United States. Its use was later expanded in 2022 to include Lennox-Gastaut syndrome, which affects roughly 48,000 people in the country. In both approved conditions, the medicine is an oral solution authorized for patients aged 2 and older.
The disorder targeted in the new study is even rarer. CDKL5 deficiency occurs in approximately one in every 40,000 to 60,000 live births and appears predominantly in females, as the responsible gene is located on the X chromosome. The gene enables the production of the CDKL5 protein, which plays a role in normal brain development.
Individuals with this disorder experience seizures starting in infancy and face substantial developmental challenges, including impairments affecting motor function, intellectual development, cortical vision, gastrointestinal function, and sleep. The median age of seizure onset is six weeks.
In its announcement, the company highlighted the daily burden on patients and caregivers. Fiona du Monceau, UCB’s executive vice president for patient evidence, stated: “Families affected by this ultra-rare condition face immense daily challenges with frequent, treatment-resistant seizures that are profoundly disruptive to daily life.” She noted that the condition continues to present an unmet need.
Among the study’s findings, patients treated with Fintepla experienced a 47.6% reduction in countable motor seizure frequency, while those receiving a placebo recorded a 2.8% reduction. Additionally, 45% of participants in the Fintepla group achieved at least a 50% decrease in seizure frequency, compared with 4.5% of those on placebo.
Investigators also observed a greater level of improvement on the Clinical Global Impression-Improvement scale, rating 38% of Fintepla-treated patients as “much improved” or “very much improved,” compared with 7% in the placebo arm. Caregiver assessments aligned with these findings, with 52% reporting significant improvement in the Fintepla group versus 2% for placebo. The trial also showed that Fintepla recipients had more seizure-free days, with a median monthly increase of 6.4 days, compared with 0.1 day for those receiving a placebo.
Fintepla was initially developed by California-based Zogenix, which obtained its first regulatory approval before being acquired by UCB for $1.9 billion. UCB reported 203 million euros in Fintepla sales for the first half of the current year, with revenue rising each year since the drug’s introduction.
The latest Phase 3 results reinforce UCB’s position as a leading developer in rare epilepsy therapies. Beyond seizure reduction, the trial highlighted improvements in patient quality of life and caregiver burden, which are critical considerations for regulatory and clinical decision-making.
Analysts expect UCB to leverage these findings for global submissions, potentially expanding Fintepla’s reach to additional countries with limited treatment options for CDKL5 deficiency. With the ultra-rare nature of the disorder, these approvals could significantly impact the daily lives of affected families.
Moreover, the study provides valuable insights for future research in ultra-rare epilepsy syndromes, encouraging further exploration of precision therapies. By demonstrating both efficacy and tolerability, UCB continues to advance the field of pediatric and rare disease neurology, underscoring its commitment to addressing unmet medical needs worldwide.
