Afamelanotide Medicine 06 June 2023 Extended indication Extension of indication for the prevention of phototoxicity in adolescent patients (12 to under 18 years of age) with erythropoietic protoporphyria (EPP). Therapeutic value Possible added value Total cost € 139,860.00 Registration phase No registration expected Product Active substance Afamelanotide Domain Metabolism and Endocrinology Reason of inclusion Indication extension IND Main indication Metabolic diseases Extended indication Extension of indication for the prevention of phototoxicity in adolescent patients (12 to under 18 years of age) with erythropoietic protoporphyria (EPP). Current proprietary name Already available biosimilars / generics Proprietary name Scenesse Manufacturer Clinuvel Portfolio holder Mechanism of action Unknown Route of administration Subcutaneous Therapeutical formulation Implant Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date February 2023 Expected Registration Orphan drug Yes Registration phase No registration expected Reimbursement Medicine sluice Additional remarks Registratieaanvraag teruggetrokken mei 2024 Therapeutic value Current treatment options Op dit moment is er nog geen behandeling. Therapeutic value Possible added value This assessment does not indicate any potential inclusion in the package. Substantiation Er is wellicht nog meer meerwaarde bij kinderen dan bij volwassen. Er is zeker effectiviteit. Er ontstaan pijnlijke blaren bij blootstelling van de huid aan zonlicht. Dit zorgt ervoor dat kinderen snel in een sociaalisolement belanden. Duration of treatment Frequency of administration Dosage per administration 16mg References NCT01605136 en NCT00979745 Debby Wensink, Margreet A.E.M. Wagenmakers & Janneke G. Langendonk (2021) Afamelanotide for prevention of phototoxicity in erythropoietic protoporphyria, Expert Review of Clinical Pharmacology, 14:2, 151-160, DOI: 10.1080/17512433.2021.1879638 Additional remarks Toediening om de 60 dagen. Expected patient volume per year Patient volume < 10 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Lecha, M., Puy, H. & Deybach, JC. Erythropoietic protoporphyria. Orphanet J Rare Dis 4, 19 (2009). https://doi.org/10.1186/1750-1172-4-19 Additional remarks Vanuit de literatuur wordt er gerekend met een prevalentie van 1 op de 75.000 tot 1 op de 200.000. Dit komt neer op 85 tot 226 patiënten in Nederland. De meeste patiënten met EPP worden al behandeld vanuit het porfyrie expertise centrum in het ErasmusMC. Schatting is dat er maximaal 10 patiënten bijkomen in de komende 3 jaar (bron expertisecentrum) Expected cost per patient per year Cost € 13,986.00 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 Gemiddelde prijs per 1 injectieflacon Scenesse, implantaat 16mg €15.370,04 inclusief btw. Potential total cost per year Total cost € 139,860.00 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