Hydroxycarbamide Medicine 06 June 2023 Extended indication Extension of indication to include the prevention of vaso-occlusive complications of sickle cell disease in children from 6 months to 2 years of age. Therapeutic value No estimate possible yet Total cost Registration phase Registration application pending Product Active substance Hydroxycarbamide Domain Cardiovascular diseases Reason of inclusion Indication extension IND Main indication Other non-oncological hematological medications Extended indication Extension of indication to include the prevention of vaso-occlusive complications of sickle cell disease in children from 6 months to 2 years of age. Current proprietary name Already available biosimilars / generics Proprietary name Xromi Manufacturer Nova Laboratories Portfolio holder Mechanism of action Unknown Route of administration Oral Therapeutical formulation Drink Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date January 2023 Expected Registration January 2024 Orphan drug No Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Er zijn op dit moment nog geen studieresultaten bekend bij patiënten onder de 2 jaar. Duration of treatment Frequency of administration Dosage per administration References NCT03763656 (HUPK) Additional remarks Expected patient volume per year Patient volume 40 - 60 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References https://www.erasmusmc.nl/nl-nl/patientenzorg/aandoeningen/sikkelcelziekte#26e2db54-b255-4e9e-997a-ef6e3e754212; RIVM, Sikkelcelziekte- Informatie in het kader van de neonatale hielprikscreening Additional remarks In Nederland zijn er ongeveer 2.000 patiënten met sikkelcelziekte. Elk jaar worden er zo’n 40-60 baby’s geboren met sikkelcelziekte. Expected cost per patient per year Cost References Medicijnkosten.nl Additional remarks Xromi, drank 100mg/ml €490,50 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