Macitentan

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
Registration phase	Registration application pending

Product

Active substance	Macitentan
Domain	Cardiovascular diseases
Reason of inclusion	Indication extension
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.
Proprietary name	Opsumit
Manufacturer	Actelion
Route of administration	Oral
Therapeutical formulation	Tablet
Budgetting framework	Extramural (GVS)
Additional remarks	Endothelin A receptor antagonists; Endothelin B receptor antagonists

Registration route	Centralised (EMA)
Type of trajectory	Normal trajectory
ATMP	No
Submission date	October 2023
Expected Registration	August 2024
Orphan drug	Yes
Registration phase	Registration application pending
Additional remarks	NCT02932410

Therapeutic value	No estimate possible yet

There is currently nothing known about the expected patient volume.

There is currently nothing known about the expected cost.

There is currently nothing known about the possible total cost.

There is currently nothing known about off label use.

There is currently nothing known about indication extensions.

There is currently no futher information available.