Ambrisentan Medicine 07 December 2021 Extended indication Extension application to introduce a new strength (2.5 mg film-coated tablet), grouped with an extension of indication to include treatment of PAH in adolescents and children (8 to less than 18 years). Therapeutic value No estimate possible yet Total cost Registration phase Registered Product Active substance Ambrisentan Domain Lung diseases Reason of inclusion Indication extension IND Main indication Lung diseases other Extended indication Extension application to introduce a new strength (2.5 mg film-coated tablet), grouped with an extension of indication to include treatment of PAH in adolescents and children (8 to less than 18 years). Current proprietary name Already available biosimilars / generics Proprietary name Volibris Manufacturer GSK Portfolio holder Mechanism of action Receptor antagonist Route of administration Oral Therapeutical formulation Tablet Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date 2021 Expected Registration September 2021 Orphan drug Yes Registration phase Registered Reimbursement Medicine sluice Additional remarks Positieve CHMP opinie juli 2021. Therapeutic value Current treatment options Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation betreft een indicatie-uitbreiding naar kinderen en adolescenten. 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 ntvg.nl Additional remarks De prevalentie van PAH in Nederland is ongeveer 15-52 per 1 miljoen inwoners. Gezien het een pediatrische uitbreiding betreft zal slecht een klein deel ven de patiënten in aanmerking komen. Expected cost per patient per year Cost References medicijnkosten.nl, GIP databank Additional remarks Prijs per tablet van 5mg per stuk €52,32 exclusief afleverkosten en inclusief BTW. Prijs per tablet van 10mg per stuk €83,70 exclusief afleverkosten en inclusief BTW. De vergoeding per gebruiker in 2020 betrof €22.243. 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 No Indication extensions References Adis Insight Additional remarks Other information Additional remarks