Afamelanotide

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
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)

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.

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.