Patisiran Medicine 09 January 2018 Extended indication Transthyretin-mediated amyloidosis (ATTR amyloidosis). Therapeutic value No judgement Total cost Registration phase Unknown Product Active substance Patisiran Domain Neurological disorders Reason of inclusion Main indication Neurological disorders other Extended indication Transthyretin-mediated amyloidosis (ATTR amyloidosis). Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Alnylam Portfolio holder Mechanism of action Unknown Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Werkingsmechanisme: siRNA gericht tegen transthyretine. Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP Unknown Submission date Expected Registration 2019 Orphan drug Yes Registration phase Unknown Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Therapeutic value No judgement 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 < 340 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References EMA Additional remarks At the time of designation, ATTR amyloidosis affected less than 0.2 in 10,000 people in the European Union (EU).' (EMA) 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