Ribociclib

Extended indication	Treatment of ambulatory Duchenne Muscular Dystrophy patients
Therapeutic value	No estimate possible yet
Total cost	975,000,000.00
Registration phase	Clinical trials

Product

Active substance	Fordadistrogene movaparvovec
Domain	Neurological disorders
Reason of inclusion	New medicine (specialité)
Main indication	Duchenne
Extended indication	Treatment of ambulatory Duchenne Muscular Dystrophy patients
Manufacturer	Pfizer
Mechanism of action	Gene therapy
Route of administration	Intravenous
Therapeutical formulation	Intravenous drip
Budgetting framework	Intermural (MSZ)

Registration

Registration route	Centralised (EMA)
ATMP	Yes
Submission date	April 2023
Expected Registration	February 2024
Orphan drug	Yes
Registration phase	Clinical trials
Additional remarks	Dit geneesmiddel is geclassificeerd als een ATMP. De Fabrikant geeft aan indiening in het tweede kwartaal van 2023 te verwachten.

Therapeutic value

Current treatment options	Steroïden.
Therapeutic value	No estimate possible yet
References	NCT04281485

Expected patient volume per year

Patient volume	< 500 Market share is generally not included unless otherwise stated.
References	Spierziekten Nederland (1);
Additional remarks	Er zijn in Nederland rond de 500 patiënten met Duchenne spierdystrofie (1).

Patient volume

< 500

Market share is generally not included unless otherwise stated.

References

Spierziekten Nederland (1);

Additional remarks

Er zijn in Nederland rond de 500 patiënten met Duchenne spierdystrofie (1).

Expected cost per patient per year

Cost	< 1,950,000.00
References	https://www.thepharmaletter.com/article/duchenne-market-to-be-an-expensive-place-to-be
Additional remarks	De prijs is nog niet vastgesteld. De prijsstelling zal niet vóór het 2de kwartaal van 2024 beschikbaar zijn. In de VS wordt verwacht dat de prijs per behandeling rond de 2 miljoen dollar zal liggen.

Cost

< 1,950,000.00

References

https://www.thepharmaletter.com/article/duchenne-market-to-be-an-expensive-place-to-be

Additional remarks

De prijs is nog niet vastgesteld. De prijsstelling zal niet vóór het 2de kwartaal van 2024 beschikbaar zijn. In de VS wordt verwacht dat de prijs per behandeling rond de 2 miljoen dollar zal liggen.

Potential total cost per year

Total cost	975,000,000.00 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

975,000,000.00

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.

Fordadistrogene movaparvovec