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Hydroxycarbamide

Xromi

Extended indication Extension of indication to include the prevention of vaso-occlusive complications of sickle cell dis
Therapeutic value No estimate possible yet
Registration phase Registration application pending

Product

Active substance Hydroxycarbamide
Domain Cardiovascular diseases
Reason of inclusion Indication extension
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.
Proprietary name Xromi
Manufacturer Nova Laboratories
Route of administration Oral
Therapeutical formulation Drink
Budgetting framework Intermural (MSZ)

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
ATMP No
Submission date January 2023
Expected Registration January 2024
Orphan drug No
Registration phase Registration application pending

Therapeutic value

Therapeutic value No estimate possible yet
Substantiation Er zijn op dit moment nog geen studieresultaten bekend bij patiënten onder de 2 jaar.
References NCT03763656 (HUPK)

Expected patient volume per year

Patient volume

40 - 60

Market share is generally not included unless otherwise stated.
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

References Medicijnkosten.nl
Additional remarks Xromi, drank 100mg/ml €490,50

Potential total cost per year

There is currently nothing known about the possible total cost.

Off label use

There is currently nothing known about off label use.

Indication extension

There is currently nothing known about indication extensions.

Other information

There is currently no futher information available.