Mitapivat

Extended indication	Pyruvate kinase deficiency in transfusion-dependent and transfusion-independent adults
Therapeutic value	Possible added value
Total cost	15,800,000.00
Registration phase	Registered

Product

Active substance	Mitapivat
Domain	Cardiovascular diseases
Reason of inclusion	New medicine (specialité)
Main indication	Other non-oncological hematological medications
Extended indication	Pyruvate kinase deficiency in transfusion-dependent and transfusion-independent adults
Proprietary name	Pyrukynd
Manufacturer	Agios
Route of administration	Oral
Budgetting framework	Extramural (GVS)
Additional remarks	Werkingsmechanisme: "First-in-class, small molecule stimulator of pyruvate kinase".

Registration

Registration route	Centralised (EMA)
Type of trajectory	Normal trajectory
ATMP	No
Submission date	July 2021
Expected Registration	November 2022
Orphan drug	Yes
Registration phase	Registered
Additional remarks	positieve opinie in september 2022

Therapeutic value

Current treatment options	Geen.
Therapeutic value	Possible added value
Substantiation	Er zijn nog geen huidige behandelingen en daardoor kan mitapivat belangrijk zijn in het verhogen van de Hb-waarde en het voorkomen van transfusies en van patiënten. De eerste eindpunten van de fase 3 studie zijn behaald. Verdere resultaten worden afgewacht.
Duration of treatment	continuous
Frequency of administration	2 times a day
Dosage per administration	5mg-50mg
References	NCT03548220

Expected patient volume per year

Patient volume	< 50 Market share is generally not included unless otherwise stated.
References	orphanet
Additional remarks	De prevalentie is ongeveer 1-9 op de 100.000. Er wordt verwacht dat er in Nederland maximaal 50 personen in aanmerking zullen komen.

Patient volume

< 50

Market share is generally not included unless otherwise stated.

References

orphanet

Additional remarks

De prevalentie is ongeveer 1-9 op de 100.000. Er wordt verwacht dat er in Nederland maximaal 50 personen in aanmerking zullen komen.

Expected cost per patient per year

Cost	< 316,000.00
References	GoodRx
Additional remarks	Mitapivat kost in Amerika gemiddeld $26.075 (28 dagen a 2x daags 5mg). Jaarlijks zullen deze kosten neerkomen op $339,906 per patiënt per jaar. Omgerekend kost het €316.000 per patiënt per jaar.

Potential total cost per year

Total cost	15,800,000.00 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

15,800,000.00

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

Indication extension	Yes
Indication extensions	Sikkelcel ziekte
References	Adisinsight
Additional remarks	Lopende fase 1 studie. Indien mitapivat geregistreerd wordt voor deze indicatie zal het patiëntvolume een aanzienlijk groter volume krijgen.

Other information

There is currently no futher information available.