Macitentan Medicine 04 June 2024 Extended indication Opsumit, as monotherapy or in combination, is indicated for the long-term treatment of pulmonary arterial hypertension (PAH) in paediatric patients aged less than 18 years and bodyweight ≥ 40 kg with WHO Functional Class (FC) II to III. Therapeutic value No estimate possible yet Total cost € 1,064,864.00 Registration phase Registered and reimbursed Product Active substance Macitentan Domain Cardiovascular diseases Reason of inclusion Indication extension IND Main indication Other medication for cardiovascular diseases Extended indication Opsumit, as monotherapy or in combination, is indicated for the long-term treatment of pulmonary arterial hypertension (PAH) in paediatric patients aged less than 18 years and bodyweight ≥ 40 kg with WHO Functional Class (FC) II to III. Current proprietary name Already available biosimilars / generics Proprietary name Opsumit Manufacturer Janssen Portfolio holder Mechanism of action Unknown Route of administration Oral Therapeutical formulation Tablet Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Endothelin A receptor antagonists; Endothelin B receptor antagonists Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date October 2023 Expected Registration September 2024 Orphan drug Yes Registration phase Registered and reimbursed Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Bosentan Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Bosentan wordt momenteel bij kinderen met PAH ingezet, net als macitentan een endotheline-antagonist, maar is minder selectief voor enthotheline-A. De therapeutische meerwaarde is niet onderzocht in de categorie >40 kg tot 78 jaar. Bij pasgeborenen is wel aangetoond in een kleine groep (n=30) dat bosentan niet-inferieur is ten opzichte van macitentan (2). Duration of treatment Frequency of administration 1 times a day Dosage per administration Afhankelijk van leeftijd of lichaamsgewicht References NCT02932410 (TOMORROW) (1); Kashaki et al. Ped Neo. 2023 (2). Additional remarks Expected patient volume per year Patient volume 7 - 56 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Expertopinie (1). Additional remarks PAH is een zeldzame aandoening. Een schatting is dat er 2 tot 16 kinderen per miljoen zijn met PAH. Er zijn circa 3,5 miljoen Nederlandse kinderen, dus dit betreft 7 tot 56 kinderen. Hierbij is nog niet het gewicht van minimaal 40kg (meestal rond 12 jaar) meegenomen (1). Expected cost per patient per year Cost € < 33,277.00 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 Opsumit tablet filmomhuld 10mg: €99,31 (inclusief BTW). Uitgaande van een doorlopende, dagelijkse behandelduur gaat dit middel €33.277 (exclusief BTW) per patiënt per jaar kosten (€99,31/109∗100∗365,24). Potential total cost per year Total cost € 1,064,864.00 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Unknown Indication extensions References Additional remarks Other information Additional remarks