Extended indication Ralinepag in combination with standard therapy for the treatment of symptomatic WHO group 1 pulmonary arterial hypertension in adults and the elderly.
Therapeutic value No estimate possible yet
Total cost 12,555,000.00
Registration phase Clinical trials

Product

Active substance Ralinepag
Domain Lung diseases
Reason of inclusion New medicine (specialité)
Main indication Lung diseases other
Extended indication Ralinepag in combination with standard therapy for the treatment of symptomatic WHO group 1 pulmonary arterial hypertension in adults and the elderly.
Manufacturer United Therapeutics Corporation
Mechanism of action Receptor agonist
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 2026
Expected Registration 2027
Orphan drug Yes
Registration phase Clinical trials
Additional remarks Ralinepag wordt onderzocht in de ADVANCE OUTCOMES fase 3-trial. United Therapeutics verwacht de resultaten van deze studie in 2026. Hierbij kan een registratie in 2027 verwacht worden.

Therapeutic value

Current treatment options Epoprostenol; treprostinil; iloprost; selexipag
Therapeutic value No estimate possible yet

This assessment does not indicate any potential inclusion in the package.

Substantiation Het is de verwachting dat ralinepag een kleine rol zal spelen binnen dit indicatiegebied. Selexipag dat qua werking vergelijkbaar is heeft tot nu toe geen goede resultaten opgeleverd. Er is weinig effect en er zijn relatief veel bijwerkingen. Verdere studieresultaten dienen nog wel afgewacht te worden.
References NCT03626688 (ROR-PH-301, ADVANCE OUTCOMES); NCT03683186 (ROR-PH-303)

Expected patient volume per year

Patient volume 288 - 522

Market share is generally not included unless otherwise stated.

References Informatie voor de huisarts over Pulmonale Hypertensie. Stichting PAH Nederland et al. 2016 (1).
Additional remarks De prevalentie van PAH in Nederland is ongeveer 16 tot 29 per miljoen inwoners (1).

Expected cost per patient per year

Cost 31,000.00

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.

Additional remarks Kosten op basis van IHSI-inschatting, afgeleid van de mediane kosten van dezelfde ATC-klasse (B01AC) geneesmiddelen in Nederland (exclusief heparine).

Potential total cost per year

Total cost 12,555,000.00

Off label use

Indication extension

Other information