Vutrisiran Medicine 07 December 2021 Extended indication Hereditary transthyretin-mediated amyloidosis. Therapeutic value No estimate possible yet Total cost Registration phase Registration application pending Product Active substance Vutrisiran Domain Neurological disorders Reason of inclusion New medicine (specialité) Main indication Neurological disorders other Extended indication Hereditary transthyretin-mediated amyloidosis. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Alnylam Portfolio holder Mechanism of action Unknown Route of administration Subcutaneous Therapeutical formulation Injection Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Systemically delivered RNA interference therapy. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date September 2021 Expected Registration October 2022 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks 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