UCB SA — Fintepla EMA EPAR public assessment report (EMEA/H/C/003933, 2020)
UCB SA · 2020 · EMA EPAR public assessment report · company profile →
Report summary
The EMA's assessment of Fintepla (fenfluramine hydrochloride) for Dravet Syndrome, despite its orphan designation, did not receive accelerated review due to concerns about efficacy relative to existing treatments and known safety risks, particularly cardiac issues. Pharmaceutical development confirmed Fintepla's stability and suitability for pediatric use. Non-clinical studies detailed its serotonin-modulating pharmacology and pharmacokinetics, with toxicology establishing a NOAEL. Clinical trials (Study 1 and Study 1504) demonstrated significant reductions in convulsive seizure frequency, meeting primary endpoints. However, safety concerns include decreased appetite, weight loss, and cardiac adverse events like valvular heart disease and pulmonary arterial hypertension, necessitating a robust risk management plan with ongoing pharmacovigilance and a long-term registry.
Key findings — cited to the page
| Irritability (placebo group) | 2% | · p.73 |
| Irritability (ZX008 groups) | 4% | · p.73 |
| Irritability incidence | 4 % | ZX008 group · p.92 |
| Irritability incidence (placebo) | 2 % | · p.92 |
| Fenfluramine concentration in oral solution | 2.5 mg/mL fenfluramine HCl | Fintepla · p.13 |
| Fenfluramine use for appetite suppression (Europe) | 50 million Europeans | Europe · p.13 |
| Fenfluramine withdrawal reason | association with cardiac valve abnormalities | world-wide · p.13 |
| Dravet Syndrome contribution to severe epilepsies | 7% | · p.12 |
| DS patients with truncating mutations | 40% | · p.12 |
| DS patients with SCN1A abnormalities | 70% and 80% | · p.12 |
| DS patients with severe intellectual impairment | 50% | · p.12 |
| Proposed indication age range | 2 years to 17 years and adults | Fintepla · p.8 |
Source: UCB SA ↗ · License: EMA - reuse permitted with acknowledgement. Summary and findings are extracted from the published document; every figure cites its page. Informational only — not investment advice. Explore the Neurology library →