Expand all sections

Riociguat

Adempas

Extended indication Adempas is indicated for the treatment of PAH in paediatric patients aged less than 18 years of age
Therapeutic value No estimate possible yet
Total cost 240,000.00
Registration phase Positive CHMP opinion

Product

Active substance Riociguat
Domain Lung diseases
Reason of inclusion Indication extension
Main indication Lung diseases other
Extended indication Adempas is indicated for the treatment of PAH in paediatric patients aged less than 18 years of age and body weight ≥ 50 kg with WHO Functional Class (FC) II to III in combination with endothelin receptor antagonists.
Proprietary name Adempas
Manufacturer Bayer
Portfolio holder Bayer
Mechanism of action Vasodilator
Route of administration Oral
Therapeutical formulation Coated tablet
Budgetting framework Extramural (GVS)

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
Submission date September 2022
Expected Registration June 2023
Orphan drug Yes
Registration phase Positive CHMP opinion
Additional remarks Positieve opinie CHMPO in april 2023.

Therapeutic value

Therapeutic value No estimate possible yet
Substantiation Het veiligheidsprofiel van riociguat lijkt acceptabel te zijn aan de hand van de studieresultaten.
References The PATENT–CHILD study. Pulm Circ. 2022 Jul; 12(3);

Expected patient volume per year

Patient volume

< 10

Market share is generally not included unless otherwise stated.
References Ntvg.nl (1); Gorenflo. Cardiovasc Diagn Ther. 2021 (2); Expertopinie (3);
Additional remarks De prevalentie van PAH in Nederland is ongeveer 15-52 per 1.000.000 inwoners. Bij kinderen is het echter vele malen zeldzamer met een incidentie van 0,7 (iPAH) en 2,2 (PAH-AHF) per miljoen kinderen (2). Er zijn ongeveer 2,5 miljoen kinderen in deze leeftijdsgroep in Nederland. Er wordt verwacht dat er niet meer dan 10 patiënten in aanmerking zullen komen (3).

Expected cost per patient per year

Cost < 24,000.00
References GIPdatabank
Additional remarks Voor de huidige indicatie werd in 2021 €24.202 per gebruiker vergoed.

Potential total cost per year

Total cost

240,000.00

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.