Macitentan Medicine 04 June 2024 Extended indication Extension of indication to include, as monotherapy or in combination, the long-term treatment of pulmonary arterial hypertension (PAH) in paediatric patients aged 1 month to less than 18 years of age of WHO Functional Class (FC) I to III. Therapeutic value No estimate possible yet Total cost Registration phase Registration application pending Product Active substance Macitentan Domain Cardiovascular diseases Reason of inclusion Indication extension IND Main indication Other medication for cardiovascular diseases Extended indication Extension of indication to include, as monotherapy or in combination, the long-term treatment of pulmonary arterial hypertension (PAH) in paediatric patients aged 1 month to less than 18 years of age of WHO Functional Class (FC) I to III. Current proprietary name Already available biosimilars / generics Proprietary name Opsumit Manufacturer Actelion 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 August 2024 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks NCT02932410 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 Frequency of administration Dosage per administration References Additional remarks Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Additional remarks Expected cost per patient per year Cost References Additional remarks Potential total cost per year Total cost 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