Fenfluramine Medicine 08 December 2020 Extended indication Extension of indication to include treatment of seizures associated with Lennox-Gastaut syndrome as an add on therapy to other anti-epileptic medicines for patients 2 years of age and older. Therapeutic value No estimate possible yet Total cost € 27,962.00 Registration phase Registered Product Active substance Fenfluramine Domain Neurological disorders Reason of inclusion Indication extension IND Main indication Epilepsy Extended indication Extension of indication to include treatment of seizures associated with Lennox-Gastaut syndrome as an add on therapy to other anti-epileptic medicines for patients 2 years of age and older. Current proprietary name Already available biosimilars / generics Proprietary name Fintepla Manufacturer UCB Portfolio holder Mechanism of action Other 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 Unknown Particularity Unknown ATMP No Submission date January 2022 Expected Registration January 2023 Orphan drug Yes Registration phase Registered Reimbursement Medicine sluice Additional remarks EMA goedkeuring januari 2023, indicatie LGS. Therapeutic value Current treatment options Valproaat (valproic acid) als monotherapie of in combinatie met lamotrigine, topiramate, rufinamide, clobazam en in de toekomst mogelijk ook canabidiol. Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation In de fase 3 studie (Study 1601) werd superioriteit ten opzichte van placebo aangetoond in reductie van het aantal convulsies en de secundaire eindpunten. Er wordt op dit moment gelijke effectiviteit verwacht met cannabidiol. Duration of treatment Frequency of administration 1 times a day Dosage per administration 0.2 or 0.8 mg/kg/day References NCT03355209; NCT03936777(Study 1601); Balagura et al. Adjunctive Rufinamide in Children with Lennox-Gastaut Syndrome: A Literature Review. Neuropsychiatr Dis Treat. 2020. Additional remarks Expected patient volume per year Patient volume 909 - 1,818 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References www.volksgezondheidenzorg.info; Asadi-Pooya et al. 2018; Additional remarks In 2016 stonden er naar schatting 181.800 mensen bij de huisarts geregistreerd met de diagnose epilepsie. De prevalentie van LGS wordt geschat tussen de 1% en 2% van alle patiënten met epilepsie. In Nederland betekent dit dat er tussen de 1.818 en 3.636 patiënten met Lennox-Gastaut zijn. Het is mogelijk dat ongeveer 50% van de patiënten met Lennox-Gastaut Syndrome zodanig "treatment-resistent" is, dat zij in aanmerking zouden komen voor een behandeling met fenfluramine. Dit zou betekenen dat er 909 tot 1.818 patiënten in Nederland in aanmerking komen. Expected cost per patient per year Cost € 1.00 - 40.00 This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year. References G-Standaard 2023 Additional remarks Min-max jaarlijkse behandelkosten voor 1 mg-26 mg. Op basis van DDD 26mg: 25.071 Euro/jaar. Potential total cost per year Total cost € 27,962.00 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions No Indication extensions References Additional remarks Geen nieuwe indicatie-uitbreidingen buiten de indicaties in de Horizonscan Geneesmiddelen. Other information Additional remarks