UCB’s FINTEPLA®▼ (fenfluramine) oral solution has received approval from the Japanese Ministry of Health, Labour, and Welfare (MHLW) for treating seizures linked with Lennox-Gastaut syndrome (LGS) as an additional therapy alongside other anti-epileptic medications for patients aged two and above.
The MHLW’s approval stems from safety and efficacy findings from a global Phase 3 clinical trial (1601 Study Cohort A & B), involving 296 LGS patients aged 2-35, including 33 from Japan. In the Japanese subset, adjunctive fenfluramine, at 0.7 mg/kg/day, demonstrated a significant reduction in drop seizure frequency compared to placebo. The most common (≥10%) non-cardiovascular treatment–emergent adverse events (TEAEs) included decreased appetite, somnolence, weight loss, diarrhea, and nasopharyngitis. Notably, no cases of valvular heart disease or pulmonary arterial hypertension were reported.
Mike Davis, Head of Global Epilepsy & Rare Syndromes at UCB, emphasized the company’s commitment to addressing the unmet needs in LGS, expressing anticipation for the positive impact of this treatment advancement in Japan. Kanako Kikuchi, Head of UCB Japan, hailed the approval as a significant achievement, aligning with UCB’s dedication to introducing innovative medicines for severe neurological and immunological conditions in Japan.
Lennox-Gastaut syndrome (LGS) is a severe childhood-onset developmental and epileptic encephalopathy (DEE), characterized by various drug-resistant seizures, significantly impacting patients’ quality of life and their families. Motor, cognitive, and behavioral abnormalities are persistent, with intellectual disability worsening over time. Seizures leading to falls (“drop attacks/seizures”) are prevalent, with tonic seizures being a hallmark feature. Generalized tonic–clonic seizures (GTCS) are commonly observed and are a primary risk factor for sudden unexpected death in epilepsy (SUDEP), posing a considerable threat to patients with LGS.
Fenfluramine is marketed in Japan by Nippon Shinyaku Co., Ltd., under an exclusive sales agreement signed in 2019 between Zogenix Inc. (acquired by UCB in 2022) and Nippon Shinyaku Co., Ltd., with UCB holding the Marketing Authorization.
