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Eplontersen

Wainzua

Extended indication

Wainzua is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (ATTRv) in a

Therapeutic value

No estimate possible yet
Registration phase

Positive CHMP opinion

Product

Active substance

Eplontersen
Domain

Neurological disorders
Reason of inclusion

New medicine (specialité)
Main indication

Neurological disorders other
Extended indication

Wainzua is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (ATTRv) in adult patients with stage 1 or stage 2 polyneuropathy.
Proprietary name

Wainzua
Manufacturer

AstraZeneca
Portfolio holder

AstraZeneca
Mechanism of action

Antisense oligonucleotide
Route of administration

Subcutaneous
Therapeutical formulation

Injection
Budgetting framework

Extramural (GVS)
Additional remarks 
Prealbumin expression inhibitor; RNA interference.

Registration

Registration route

Centralised (EMA)
ATMP

No
Submission date

October 2023
Expected Registration

January 2025
Orphan drug

Yes
Registration phase

Positive CHMP opinion
Additional remarks 
Positieve CHMP opinie oktober 2024

Therapeutic value

Current treatment options

Huidige behandelopties zijn: diflunisal en tafamidis. Daarnaast zal het middel moeten gaan concurreren met patisiran en inotersen.
Therapeutic value

No estimate possible yet
Substantiation

Eplontersen toonde effectiviteit aan in de fase 3 trial voor hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) na 66 weken. n=166, persbericht: er werd een relevante verandering gezien in de 'modified Neuropathy Impairment Score'.
Duration of treatment

continuous
Frequency of administration

1 times every 4 weeks
Dosage per administration

45mg
References 
NCT04136184
Additional remarks 
Vergelijkende studie met inotersen (toediening eens per week).

Expected patient volume per year

Patient volume

30 - 60

Market share is generally not included unless otherwise stated.
Additional remarks 
Er zijn volgens een persbericht van AstraZeneca 40.000 patiënten met ATTRv-PN wereldwijd. Op dit moment ontvangen er 29 patiënten patisiran. De verwachting is dat er mogelijk 30 tot 60 patiënten in aanmerking zullen gaan komen.

Expected cost per patient per year

Additional remarks 
lijst prijs is nog niet bekend.

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

Indication extension

Yes
Indication extensions

ATTR Cardiomyopathy (CM)
References 
Fabrikant
Additional remarks 
Deze indicatie wordt in 2027 verwacht.

Other information

There is currently no futher information available.