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 comments
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 comments
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 comments
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.

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.