Crinecerfont Medicine 03 June 2025 Extended indication Congenital Adrenal Hyperplasia 2 Years to 17 Years (Child ). Therapeutic value No estimate possible yet Total cost Registration phase Clinical trials Product Active substance Crinecerfont Domain Metabolism and Endocrinology Reason of inclusion New medicine (specialité) Main indication Metabolic diseases Extended indication Congenital Adrenal Hyperplasia 2 Years to 17 Years (Child ). Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Neurocrine Biosciences Portfolio holder Mechanism of action Unknown Route of administration Oral Therapeutical formulation Capsule Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP No Submission date December 2025 Expected Registration January 2026 Orphan drug No Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks In behandeling bij de FDA. Registratie bij de FDA wordt verwacht einde 2024. Therapeutic value Current treatment options Standaard glucocorticoid therapie: hydrocortisone, prednisone, prednisolone, dexamethasone. Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration 2 times a day Dosage per administration References NCT04806451 Additional remarks Expected patient volume per year Patient volume 1,500 - 2,000 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References ntvg; Additional remarks Het totale aantal patiënten met bijnierinsufficiëntie ligt vermoedelijk tussen de 2.000 en 4.000. Er wordt verwacht dat er substitutie van plenadren plaats zal vinden en nieuwe patiënten gebruik zullen maken van hydrocortison. Dit zal leiden tot een mogelijk patiëntvolume tussen de 1.500 en 2.000 patiënten. Expected cost per patient per year Cost References Additional remarks Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Unknown Indication extensions References Additional remarks Other information Additional remarks