Cyclodextrine (Hydroxypropyl betadex) Medicine 09 January 2018 Extended indication Ziekte van Niemann-Pick, type C Therapeutic value No judgement Total cost Registration phase Clinical trials Product Active substance Cyclodextrine (Hydroxypropyl betadex) Domain Metabolism and Endocrinology Reason of inclusion Main indication Metabolic diseases Extended indication Ziekte van Niemann-Pick, type C Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Vtesse Portfolio holder Mechanism of action Unknown Route of administration Intrathecal Therapeutical formulation Injection Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity New medicine ATMP Unknown Submission date Expected Registration Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration Dosage per administration References Additional remarks Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Erfelijkheid.nl; Vanier et al. Orphanet J Rare Dis. 2010 Jun 3;5:16 Additional remarks De incidentie van Niemann-Pick type C is 1:250.000, wat neerkomt op 68 patiënten in Nederland. De incidentie bij pasgeborenen is 1:120.000, dit is dus 1-2 nieuwe patiënten per jaar 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