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 comments
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.