Setmelanotide Medicine 07 December 2021 Extended indication IMCIVREE is indicated for the treatment of obesity and the control of hunger associated with genetically confirmed Bardet‑Biedl syndrome (BBS), loss-of-function biallelic pro-opiomelanocortin (POMC), including PCSK1, deficiency or biallelic leptin receptor (LEPR) deficiency in adults and children 6 years of age and above. Therapeutic value No estimate possible yet Total cost Registration phase Registered and reimbursed Product Active substance Setmelanotide Domain Metabolism and Endocrinology Reason of inclusion Indication extension IND Main indication Other metabolism and Endocrinology Extended indication IMCIVREE is indicated for the treatment of obesity and the control of hunger associated with genetically confirmed Bardet‑Biedl syndrome (BBS), loss-of-function biallelic pro-opiomelanocortin (POMC), including PCSK1, deficiency or biallelic leptin receptor (LEPR) deficiency in adults and children 6 years of age and above. Current proprietary name Already available biosimilars / generics Proprietary name Imcivree Manufacturer Rhythm Portfolio holder Mechanism of action Unknown Route of administration Subcutaneous Therapeutical formulation Injection Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date October 2021 Expected Registration September 2022 Orphan drug Yes Registration phase Registered and reimbursed Reimbursement Medicine sluice Additional remarks Positieve CHMP opinie ontvangen juli 2022. Therapeutic value Current treatment options 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 3 mg References NCT03746522 Additional remarks Expected patient volume per year Patient volume < 300 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References www.orpha.net Additional remarks In de Verenigde Staten wordt de prevalentie geschat op 1/100.000. Epidemiologische gegevens zijn beperkt in Europa, maar in Denemarken werd de prevalentie geschat op 1/59.000, en op Réunion, Frankrijk, op 1/45.000-66.000 (als gevolg van een stichter effect). Dit zou voor Nederland betekenen dat er maximaal 300 patiënten met deze ziekte leven. Expected cost per patient per year Cost References Goodrx; Additional remarks De prijs in de Verenigde Staten is gemiddeld $1,935.63 voor 4 prefilled 0,25mg pens of 0,25mg/0,5ml. De prijs in Nederland zal mogelijk lager zijn. 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