'Fintepla' reduces seizure freq…treating Dravet syndrome"

Can be used as an add-on therapy in patients aged 2 years and older

Clinical trials demonstrated a reduction of seizure frequency up to 65%...near-complete seizure freedom was confirmed

Changes are emerging in the treatment landscape for Dravet syndrome, an ultra-rare pediatric refractory disease. With the domestic approval of a serotonin-based novel drug that has demonstrated efficacy in reducing seizure frequency, expectations are high for a shift in the treatment paradigm for an area with significant unmet medical needs.

On the 4th, UCB Korea held a press conference at the Plaza Hotel in Jung-gu, Seoul, to celebrate the approval of 'Fintepla (fenfluramine),' a treatment for Dravet syndrome, in Korea.

Professor Hunmin Kim of Seoul National University Bundang Hospital 

Fintepla was designated as a drug for the second pilot project of the 'Approval-Evaluation-Negotiation Linkage System' in December 2024. This drug was officially approved in Korea last December, one year after its designation.

The specific indication is an add-on therapy for the treatment of seizures in patients with Dravet syndrome aged 2 years and older. Given the nature of pediatric patients who may have difficulty swallowing pills, Fintepla is formulated as a syrup.

Fintepla is a treatment that reduces seizures by stimulating multiple 5-HT receptor subtypes through serotonin release. It can decrease a patient's seizures through a dual mechanism that simultaneously activates serotonin receptors and sigma-1 receptors.

Fintepla's clinical value was demonstrated in three randomized Phase 3 trials (STUDY 1–3).

The analysis results of STUDY 1, which initially enrolled 119 patients, and STUDY 3, which included subsequently recruited patients, showed that the monthly average convulsive seizure frequency (MCSF) in the Fintepla-treated groups decreased by 62.3% and 64.8%, respectively. Notably, near-complete seizure freedom was confirmed only in the Fintepla-treated group.

In STUDY 2, which consisted of a 6-week baseline, 3-week titration, and 12-week maintenance period for a total of 15 weeks, patients were randomly assigned 1:1 to either Fintepla or a placebo as an add-on to the existing standard of care, stiripentol (+ clobazam and/or valproate).

In that study, the proportion of patients who showed a 50% or greater reduction in MCSF from baseline was 53.5% in the Fintepla combination group, compared with only 2% in the placebo group.

In a 3-year open-label extension study, the reduction in seizure frequency with Fintepla was maintained. 64.2% of all patients showed a 50% or greater decrease in MCSF from baseline.

In terms of safety, 216 patients with Dravet syndrome who received Fintepla showed a similar adverse event profile. The most commonly reported adverse events were decreased appetite (34.7%), diarrhea (19.9%), fatigue (19.0%), fever (18.7%), reduced blood glucose (14.4%), and somnolence (13.9%).

Professor Hunmin Kim of Seoul National University Bundang Hospital explained, "Fintepla is a treatment that has consistently proven its seizure control effect and therapeutic value in multiple clinical trials, despite the difficult environment for patient recruitment due to the nature of ultra-rare pediatric severe refractory diseases."

Professor Kim added, "Not only the mortality rate due to sudden death but also the all-cause mortality rate appeared lower in the Fintepla-treated group compared to values reported in existing literature."

Limited treatment environment for Dravet Syndrome... Expectations↑ with new treatment option

Professor Hoon-Chul Kang of the Department of Pediatric Neurology at Severance Children's Hospital

Dravet syndrome is a type of pediatric epilepsy that occurs in infancy. According to experts, the majority (80%) of this disease is caused by a mutation in the SCN1A gene.

The disease develops around 12 months of age, and up to 15% of patients die during early childhood or adolescence. Dravet syndrome patients are at high risk for physical and mental comorbidities, such as physical stiffness, language development disorders, autism, intellectual disability, and ADHD.

Caregivers also endure high caregiving stress and low quality of life due to 24-hour care burdens, career interruptions, and loss of income.

Frequent long-term seizures in Dravet syndrome patients not only lower the quality of life for both patients and caregivers but also carry a risk of Sudden Unexpected Death in Epilepsy (SUDEP). Therefore, the primary goal of treatment is to reduce or stop seizures.

However, existing anti-epileptic drugs have limitations in controlling seizures, and some medications can even worsen them, leaving a significant unmet need in the domestic treatment environment.

Professor Hoon-Chul Kang of the Department of Pediatric Neurology at Severance Children's Hospital stated, "Treatment for Dravet syndrome should aim beyond reducing seizure frequency to managing non-seizure comorbidities and minimizing drug side effects, but this has been difficult to achieve in the current treatment environment."

Professor Kang emphasized, "There is an unmet need for treatment options that can more effectively control seizures, reduce cognitive decline and long-term disability, and improve the quality of life for patients and caregivers. Fintepla functions to prevent seizures. Seizure-freedom effects can also be expected. As it works differently from existing drugs, expectations are high."