Marstacimab

Extended indication	Voxzogo is indicated for the treatment of achondroplasia in patients 4 months of age and older whose epiphyses are not closed. The diagnosis of achondroplasia should be confirmed by appropriate genetic testing.
Therapeutic value	No estimate possible yet
Total cost	1,575,000.00
Registration phase	Registered

Product

Active substance	Vosoritide
Domain	Metabolism and Endocrinology
Reason of inclusion	Indication extension
Main indication	Other metabolism and Endocrinology
Extended indication	Voxzogo is indicated for the treatment of achondroplasia in patients 4 months of age and older whose epiphyses are not closed. The diagnosis of achondroplasia should be confirmed by appropriate genetic testing.
Proprietary name	Voxzogo
Manufacturer	BioMarin
Portfolio holder	BioMarin
Route of administration	Subcutaneous
Therapeutical formulation	Injection
Budgetting framework	Extramural (GVS)
Centre of expertise	UMC Utrecht Skeletal Dysplasia unit. Erasmus Rotterdam(Toekomstig).

Registration

Registration route	Centralised (EMA)
Type of trajectory	Normal trajectory
ATMP	No
Submission date	December 2022
Expected Registration	October 2023
Orphan drug	Yes
Registration phase	Registered
Additional remarks	Positieve CHMP-opinie in september 2023.

Therapeutic value

Current treatment options	Op dit moment is er geen behandeling voor achondroplasia. Vosoritide is wel geregistreerd voor patiënten ouder dan 2 jaar.
Therapeutic value	No estimate possible yet
Substantiation	Mogelijk is er meer winst als er al onder de twee jaar begonnen wordt met de behandeling en daardoor mogelijk een gunstigere skeletgroei. Daarnaast is er mogelijk een gunstig effect op het voorkomen van vernauwing van het wervelkanaal.
Frequency of administration	1 times a day
Dosage per administration	15·0 μg/kg
References	Klinisch genetica NL, Lancet September 05, 2020, Genetics in Medicine volume 23, pages 2443–2447 (2021) EMA web site
Additional remarks	Gemiddelde eindlengte is voor mannen 1 meter 31, voor vrouwen 1 meter 24. Vosoritide geeft groei van 1.57cm per jaar en verbetering van lichaamsproporties. Onderzocht in leeftijdsgroep 5 tot 17 jaar. Beste effect op groei tussen 2 tot 5 jaar. De vraag is of dat het middel ook goed werkt tijdens groeispurt omdat deze afwijkend is bij achondroplasie.

Expected patient volume per year

Patient volume	7 Market share is generally not included unless otherwise stated.
References	Birth prevalence of achondroplasia: A systematic literature review and meta‐analysis. Pamela K. Foreman. Am J Med Genet A. 2020 (1);
Additional remarks	De geboorteprevalentie is 5 op de 100.000. Dit betekent dat er 7 nieuwe patiënten per jaar in aanmerking komen (1).

Patient volume

7

Market share is generally not included unless otherwise stated.

References

Birth prevalence of achondroplasia: A systematic literature review and meta‐analysis. Pamela K. Foreman. Am J Med Genet A. 2020 (1);

Additional remarks

De geboorteprevalentie is 5 op de 100.000. Dit betekent dat er 7 nieuwe patiënten per jaar in aanmerking komen (1).

Expected cost per patient per year

Cost	200,000.00 - 250,000.00
References	https://www.bvkm.nl/thema/achondroplasie-en-vosoritide-veelgestelde-vragen-en-antwoorden/
Additional remarks	De fabrikant heeft in eerste instantie ingezet op gemiddeld €200.000 tot € 250.000 per patiënt per jaar.

Potential total cost per year

Total cost	1,575,000.00 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.

Total cost

1,575,000.00

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.

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.

Vosoritide