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Vutrisiran

Extended indication Hereditary transthyretin-mediated amyloidosis.
Therapeutic value No estimate possible yet
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.
Manufacturer Alnylam
Route of administration Subcutaneous
Therapeutical formulation Injection
Budgetting framework Intermural (MSZ)
Additional remarks Systemically delivered RNA interference therapy.

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
ATMP No
Submission date September 2021
Expected Registration October 2022
Orphan drug Yes
Registration phase Registration application pending

Therapeutic value

Therapeutic value No estimate possible yet

Expected patient volume per year

There is currently nothing known about the expected patient volume.

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.