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Mitapivat

Pyrukynd

Extended indication Mitapivat monotherapy for treatment of alpha or beta thalassaemia in adults and elderly
Therapeutic value No estimate possible yet
Total cost 5,640,000.00
Registration phase Clinical trials

Product

Active substance Mitapivat
Domain Cardiovascular diseases
Reason of inclusion Indication extension
Main indication Other non-oncological hematological medications
Extended indication Mitapivat monotherapy for treatment of alpha or beta thalassaemia in adults and elderly
Proprietary name Pyrukynd
Manufacturer Agios
Route of administration Oral
Therapeutical formulation Tablet
Budgetting framework Extramural (GVS)

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
ATMP No
Submission date December 2024
Expected Registration July 2025
Registration phase Clinical trials
Additional remarks Indieningsdatum en verwachte registratie op basis van IHSI-inschatting.

Therapeutic value

Current treatment options TDT: Bloedtransfusies, ijzerchelatie en luspatercept.
Therapeutic value No estimate possible yet
Substantiation Er zijn nog geen resultaten bekend van de fase 3 ENERGIZE en ENERGIZE-T studie.
Frequency of administration 2 times a day
Dosage per administration 100 mg
References NCT04770753 (ENERGIZE); NCT04770779 (ENERGIZE-T)

Expected patient volume per year

Patient volume

20

Market share is generally not included unless otherwise stated.
References Expertopinie (1)
Additional remarks Er zijn mogelijk rond de 100 transfusie-afhankelijke alfa- of bèta-thalassemie patiënten (TDT) en 100 niet-transfusie-afhankelijke alfa- of bèta-thalassemie patiënten (NTDT), het exacte aantal is niet bekend. Een ruwe inschatting is dat er 20 patiënten zijn (TDT en NTDT samen) die langdurig op mitapivat zullen staan (1).

Expected cost per patient per year

Cost 282,000.00
References GoodRX
Additional remarks Een 28 daags voorraad van 5mg per tablet is in de Verenigde Staten $25.664.97. Jaarlijks zullen deze kosten neerkomen op $307,906 per patiënt per jaar. Omgerekend kost het €282.000 per patiënt per jaar. Mogelijk zullen de kosten in Nederland lager uitvallen.

Potential total cost per year

Total cost

5,640,000.00

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.