Extended indication Pyruvate kinase deficiency in transfusion-dependent and transfusion-independent adults
Therapeutic value Possible added value
Registration phase Clinical trials

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
Manufacturer Agios
Route of administration Oral
Budgetting framework Extramural (GVS)
Additional comments Werkingsmechanisme: "First-in-class, small molecule stimulator of pyruvate kinase"

Registration

Registration route Centralised (EMA)
ATMP No
Submission date 2021
Expected Registration 2022
Orphan drug Yes
Registration phase Clinical trials
Additional comments Verwachte indieningsdatum op basis van primary completion datum fase 3 studie in oktober 2020.

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.
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 comments 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

There is currently nothing known about the expected cost.

Potential total cost per year

There is currently nothing known about the possible total cost.

Off label use

There is currently nothing known about off label use.

Indication extension

Indication extension Yes
Indication extensions Sikkelcel ziekte
References Adisinsight
Additional comments 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.