Fenfluramine Medicine 04 June 2026 Extended indication CDKL5 syndroom Therapeutic value Total cost Registration phase Clinical trials Product Active substance Fenfluramine Domain Neurological disorders Reason of inclusion Indication extension IND Main indication Epilepsy Extended indication CDKL5 syndroom Current proprietary name Already available biosimilars / generics Proprietary name Fintepla Manufacturer UCB Portfolio holder UCB Mechanism of action Receptor agonist Route of administration Oral Therapeutical formulation Drink Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Serotonin receptor agonists; Serotonin release stimulants; Serotonin uptake inhibitors; Sigma-1 receptor agonists. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity ATMP No Submission date June 2026 Expected Registration January 2027 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Verwachte registratie op basis inschatting van fabrikant. Therapeutic value Current treatment options Bij jonge kinderen wordt vaak gebruikt vigabatrine of fenobarbital. Bij oudere kinderen worden medicijnen gebruikt zoals natriumvalproaat, levetiracetam, clobazam en zonisamide. Therapeutic value Substantiation Studieresultaten nog niet beschikbaar Duration of treatment Frequency of administration 2 times a day Dosage per administration 0.8 mg/kg/dag References Kinderneurologie.eu; rarediseases.org; NCT03861871; NCT05064878; Devinsky et al. Epilepsia. 2021. Additional remarks ʼTopline resultsʼ verwacht tweede helft van 2024. Nog geen gegevens beschikbaar op lokaal niveau (januari 2025). Expected patient volume per year Patient volume < 100 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References kinderneurologie.eu; Expertopinie Additional remarks Wereldwijd hebben iets meer dan 500 mensen deze diagnose gekregen. In Nederland zijn er rond de 20 tot 30 kinderen met CDKL5 syndroom. Een klein deel hiervan zal in aanmerking komen voor fenfluramide. Expected cost per patient per year Cost References Additional remarks Fenfluramine is opgenomen in de G-Standaard. Onderdeel financieel arrangement lopende tot 1/1/2030 (staatscourant). Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use Indications off label use References Additional remarks Indication extension Indication extensions Indication extensions References Additional remarks Other information Additional remarks