Vutrisiran Medicine 03 December 2024 Extended indication Extension of indication to include treatment of wild-type or hereditary transthyretin-mediated amyloidosis in adult patients with cardiomyopathy (ATTR-CM) Therapeutic value No estimate possible yet Total cost Registration phase Registration application pending Product Active substance Vutrisiran Domain Neurological disorders Reason of inclusion Indication extension IND Main indication Neurological disorders other Extended indication Extension of indication to include treatment of wild-type or hereditary transthyretin-mediated amyloidosis in adult patients with cardiomyopathy (ATTR-CM) Current proprietary name Already available biosimilars / generics Proprietary name Amvuttra Manufacturer Alnylam Portfolio holder Mechanism of action Unknown Route of administration Subcutaneous Therapeutical formulation Injection 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 November 2024 Expected Registration September 2025 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Verwachte registratie op basis van 10 maanden gemiddelde doorlooptijd van indicatieuitbreiding. 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 1 times every 3 months Dosage per administration 25 mg References NCT04153149 (HELIOS-B) 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 € 397,000.00 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 FK (1) Additional remarks Prijs Amvuttra injvlst 50 mg/ml (0,5 ml): €108.182,50 (incl. BTW) (1). Als 100 mg per jaar nodig is kost een jaarlijkse behandeling €397.000,- (excl. BTW). 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