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Patisiran

Extended indication Transthyretin-mediated amyloidosis (ATTR amyloidosis).
Therapeutic value No judgement

Product

Active substance Patisiran
Domain Neurological disorders
Main indication Neurological disorders other
Extended indication Transthyretin-mediated amyloidosis (ATTR amyloidosis).
Manufacturer Alnylam
Route of administration Intravenous
Therapeutical formulation Intravenous drip
Budgetting framework Intermural (MSZ)
Additional remarks Werkingsmechanisme: siRNA gericht tegen transthyretine.

Registration

Registration route Centralised (EMA)
Expected Registration 2019
Orphan drug Yes

Therapeutic value

Therapeutic value No judgement
Duration of treatment Not found

Expected patient volume per year

Patient volume

< 340

Market share is generally not included unless otherwise stated.
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

There is currently nothing known about the expected cost.

Potential total cost per year

There is currently nothing known about the possible total cost.

Off label use

There is currently nothing known about off label use.

Indication extension

There is currently nothing known about indication extensions.

Other information

There is currently no futher information available.