Setmelanotide Medicine 04 June 2024 Extended indication Extension of indication to include the population of children aged 2 years and above for the treatment of pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin Type 1 (PCSK1) deficiency or biallelic leptin receptor (LEPR) deficiency and Bardet-Biedl Syndrome (BBS) for IMCIVREE. Therapeutic value No estimate possible yet Total cost € 1,875,000.00 Registration phase Registered Product Active substance Setmelanotide Domain Metabolism and Endocrinology Reason of inclusion Indication extension IND Main indication Other metabolism and Endocrinology Extended indication Extension of indication to include the population of children aged 2 years and above for the treatment of pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin Type 1 (PCSK1) deficiency or biallelic leptin receptor (LEPR) deficiency and Bardet-Biedl Syndrome (BBS) for IMCIVREE. 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 December 2023 Expected Registration September 2024 Orphan drug Yes Registration phase Registered Reimbursement Medicine sluice Additional remarks Positieve CHMP opinie juni 2024 Therapeutic value Current treatment options Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation NCT04966741 is een open label studie waarbij er een 25% afname in BMI was in de POMC/PCSK1/LEPR-deficiëntiegroep en een 10% afname in de BBS-groep. Dit is een redelijke effectiviteit. Duration of treatment Frequency of administration Dosage per administration References NCT04966741 Additional remarks 0,5 mg once daily for 52 weeks. The dose was escalated by increments of 0.5 mg every 2 weeks, if tolerated, at the dose escalation visits (Weeks 2, 4, and 6) to a maximum dose of 0.5 to 2.0 mg QD with the maximum dose based on body weight. Expected patient volume per year Patient volume 10 - 20 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Horizonscan Geneesmiddelen setmelanotide. 2022 (1); Expertopinie (2). 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 op 59.000, en op Réunion (Frankrijk) 1 op 45.000 tot 66.000 (als gevolg van een stichter effect). Dit zou voor Nederland betekenen dat er maximaal 300 patiënten met deze ziekte leven (1). De indicatieuitbreiding van 2 tot 6 jaar oud zal een geringe invloed hebben op het patiëntvolume. Het zal om ongeveer 10 tot 20 patiënten gaan (2). Expected cost per patient per year Cost € 50,000.00 - 200,000.00 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 medicijnkosten.nl Additional remarks De gemiddelde prijs van een injectieflacon IMCIVREE, 1 ml INJECTIEVLOEISTOF 10 mg/ml is € 2.997,50 incl btw. In de klinische studie wordt een dosis van 0,5 tot 2 mg genoemd. Potential total cost per year Total cost € 1,875,000.00 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