Extended indication Extension of indication for the prevention of phototoxicity in adolescent patients (12 to under 18 y
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
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).
Proprietary name Scenesse
Manufacturer Clinuvel
Route of administration Subcutaneous
Therapeutical formulation Implant
Budgetting framework Intermural (MSZ)

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
ATMP No
Submission date February 2023
Orphan drug Yes
Registration phase No registration expected
Additional remarks Registratieaanvraag teruggetrokken mei 2024

Therapeutic value

Current treatment options Op dit moment is er nog geen behandeling.
Therapeutic value Possible added value
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 continuous
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.

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
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

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.

Off label use

There is currently nothing known about off label use.

Indication extension

There is currently nothing known about indication extensions.

Other information

There is currently no futher information available.