Macitentan Medicine 12 June 2019 Extended indication Extension of Indication to include treatment of patients with inoperable chronic thromboembolic pulmonary hypertension (CTEPH) Therapeutic value No judgement Total cost € 4,320,000.00 Registration phase Registration application pending Product Active substance Macitentan Domain Cardiovascular diseases Reason of inclusion Indication extension IND Main indication Antithrombotic medications Extended indication Extension of Indication to include treatment of patients with inoperable chronic thromboembolic pulmonary hypertension (CTEPH) Current proprietary name Already available biosimilars / generics Proprietary name Opsumit Manufacturer Actelion Portfolio holder Mechanism of action Receptor antagonist Route of administration Oral Therapeutical formulation Tablet Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Endothelin receptor antagonist. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date September 2018 Expected Registration August 2019 Orphan drug No Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Orphan drug status in US for pulmonary hypertension Therapeutic value Current treatment options Riociguat Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation De verwachting is dat er substitutie van riociguat zal plaatsvinden. Duration of treatment Frequency of administration 1 times a day Dosage per administration 10 mg References NCT02060721, NCT02021292, NCT02565030 Additional remarks Expected patient volume per year Patient volume < 108 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References GIPdatabank Additional remarks In 2017 werden 108 mensen behandeld met riociguat (Adempas). Aangezien de verwachting is dat er substitutie zal plaatsvinden is dit het maximale aantal patiënten wat in aanmerking zal komen voor deze behandeling. Expected cost per patient per year Cost € 40,000.00 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. References Medicijnkosten.nl Additional remarks De prijs voor een tablet van 10 mg is €109,88. Uitgaande van een behandelduur van een jaar komt dit neer op ongeveer €40.000. Potential total cost per year Total cost € 4,320,000.00 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Yes Indication extensions Fase 3: Congenital heart defects; Eisenmenger complex References Adis Insight Additional remarks Other information Additional remarks