Sirolimus Medicine 07 June 2022 Extended indication Hyftor is indicated for the treatment of facial angiofibroma associated with tuberous sclerosis complex in adults and paediatric patients aged 6 years and older. Therapeutic value No estimate possible yet Total cost Registration phase Registered Product Active substance Sirolimus Domain Chronic immune diseases Reason of inclusion New medicine (specialité) Main indication Skin diseases Extended indication Hyftor is indicated for the treatment of facial angiofibroma associated with tuberous sclerosis complex in adults and paediatric patients aged 6 years and older. Current proprietary name Already available biosimilars / generics Proprietary name Hyftor Manufacturer Plusultra Portfolio holder Plusultra Mechanism of action Unknown Route of administration Cutaneous Therapeutical formulation Gel Budgetting framework Extramural (GVS) Centre of expertise Additional remarks mTOR remmer Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity New therapeutical formulation ATMP No Submission date December 2021 Expected Registration May 2023 Orphan drug Yes Registration phase Registered Reimbursement Medicine sluice Additional remarks Positieve CHMP-opinie in februari 2023. Therapeutic value Current treatment options Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Dit product heeft dezelfde werkzame stof als Rapamune, maar een andere toedieningsvorm. 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 Additional remarks Expected cost per patient per year Cost € > 1,700.00 This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year. References Drugs.com Additional remarks Sirolimus is in Nederland op dit moment nog enkel beschikbaar in tablet- en drankvorm. De kosten voor de topicale crème 1,5% bedragen rond de $1.852 voor een hoeveelheid van 60 gram in de Verenigde Staten (omgerekend ongeveer €1.700). De kosten in Nederland zijn nog niet bekend. 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