Vutrisiran

Extended indication	Extension of indication to include treatment of wild-type or hereditary transthyretin-mediated amyloidosis in adult patients with cardiomyopathy (ATTR-CM)
Therapeutic value	No estimate possible yet
Registration phase	Registration application pending

Product

Active substance	Vutrisiran
Domain	Neurological disorders
Reason of inclusion	Indication extension
Main indication	Neurological disorders other
Extended indication	Extension of indication to include treatment of wild-type or hereditary transthyretin-mediated amyloidosis in adult patients with cardiomyopathy (ATTR-CM)
Proprietary name	Amvuttra
Manufacturer	Alnylam
Route of administration	Subcutaneous
Therapeutical formulation	Injection
Budgetting framework	Extramural (GVS)

Registration route	Centralised (EMA)
Type of trajectory	Normal trajectory
ATMP	No
Submission date	November 2024
Expected Registration	September 2025
Orphan drug	Yes
Registration phase	Registration application pending
Additional remarks	Verwachte registratie op basis van 10 maanden gemiddelde doorlooptijd van indicatieuitbreiding.

Therapeutic value	No estimate possible yet
Duration of treatment	continuous
Frequency of administration	1 times every 3 months
Dosage per administration	25 mg
References	NCT04153149 (HELIOS-B)

There is currently nothing known about the expected patient volume.

Cost	397,000.00
References	FK (1)
Additional remarks	Prijs Amvuttra injvlst 50 mg/ml (0,5 ml): €108.182,50 (incl. BTW) (1). Als 100 mg per jaar nodig is kost een jaarlijkse behandeling €397.000,- (excl. BTW).

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.