Dipalmitoyl Hydroxyproline Medicine 03 June 2025 Extended indication Dipalmitoyl hydroxyproline monotherapy for treatment of Netherton's syndrome in adolescents over 14 years of age, adults and elderly. Therapeutic value No estimate possible yet Total cost Registration phase Clinical trials Product Active substance Dipalmitoyl Hydroxyproline Domain Chronic immune diseases Reason of inclusion New medicine (specialité) Main indication Skin diseases Extended indication Dipalmitoyl hydroxyproline monotherapy for treatment of Netherton's syndrome in adolescents over 14 years of age, adults and elderly. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Quoin Portfolio holder Mechanism of action Unknown Route of administration Cutaneous Therapeutical formulation Emulsion for cutaneous use 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 2027 Orphan drug No Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Er is geen behandeling voor het syndroom van Netherton. Het belangrijkste is dat de huid goed verzorgd wordt, zodat de kans op infecties verkleind wordt. Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration 1 times a day Dosage per administration References NCT05521438; NCT05789056 Additional remarks 2% QRX003, eenmaal daags 's ochtends gedurende 12 weken -of- 4% QRX003, eenmaal daags 's ochtends of tweemaal daags gedurende 12 weken. Expected patient volume per year Patient volume 90 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Erfelijkheid.nl Additional remarks Het Netherton syndroom komt bij ongeveer 1 op 200.000 mensen voor. 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