Riociguat Medicine 03 June 2025 Extended indication Adempas is indicated for the treatment of PAH in paediatric patients aged 6 to less than 18 years with WHO Functional Class (FC) II to III in combination with endothelin receptor antagonists (see section 5.1). Therapeutic value No estimate possible yet Total cost Registration phase Registered Product Active substance Riociguat Domain Lung diseases Reason of inclusion Indication extension IND Main indication Lung diseases other Extended indication Adempas is indicated for the treatment of PAH in paediatric patients aged 6 to less than 18 years with WHO Functional Class (FC) II to III in combination with endothelin receptor antagonists (see section 5.1). Current proprietary name Already available biosimilars / generics Proprietary name Adempas Manufacturer MSD Portfolio holder Mechanism of action Vasodilator Route of administration Oral Therapeutical formulation Coated tablet Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Dit betreft een aanpassing aan het pediatrische label van patiënten jonger dan 18 jaar en minder dan 50kg naar jonger dan 18 jaar en ouder dan 6 jaar. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity ATMP No Submission date June 2024 Expected Registration June 2025 Orphan drug Yes Registration phase Registered Reimbursement Reimbursed from the basic package Medicine sluice Additional remarks Positieve CHMP-opinie in april 2025. Therapeutic value Current treatment options Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Maximal 24 week / weeks Frequency of administration 3 times a day Dosage per administration 1,0 - 2,5 mg References NCT02562235 Additional remarks Tot 2,5mg driemaal daags (titratie tussen 1,0mg en 2,5mg) gedurende maximaal acht weken tijdens de individuele dosistitratiefase (IDT). Gevolgd door de laatste dosis toegediend in de IDT-fase gedurende maximaal 16 weken tijdens de onderhoudsfase. Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Additional remarks Deze aanpassing aan het label zal een zeer gering effect hebben op het patiëntvolume. Expected cost per patient per year Cost € < 13,800.00 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. References Medicijnkosten.nl (1). Additional remarks Een tablet Adempas 2,5mg kost €29,70 (inclusief BTW) (1). Een behandeling driemaal daags 2,5mg voor 24 weken kost €13.733 (exclusief BTW). Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use Indications off label use References Additional remarks Indication extension Indication extensions Indication extensions References Additional remarks Other information Additional remarks