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 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	No estimate possible yet

There is currently nothing known about the expected patient volume.

There is currently nothing known about the expected cost.

There is currently nothing known about the possible total cost.

There is currently nothing known about off label use.

There is currently nothing known about indication extensions.

There is currently no futher information available.