Extended indication Primary systemic amyloidosis (AL amyloïdose).
Therapeutic value No judgement
Registration phase Withdrawn

Product

Active substance Neod001
Domain Metabolism and Endocrinology
Main indication Metabolic diseases
Extended indication Primary systemic amyloidosis (AL amyloïdose).
Manufacturer Prothena
Mechanism of action Unknown
Route of administration Intravenous
Therapeutical formulation Intravenous drip
Budgetting framework Intermural (MSZ)
Additional remarks Monoklonaal antilichaam gericht tegen amyloïden

Registration

Registration route Centralised (EMA)
Type of trajectory Unknown
Particularity New medicine
ATMP Unknown
Expected Registration 2020
Orphan drug Yes
Registration phase Withdrawn
Additional remarks Ontwikkeling is gestopt per april 2018. Primaire eindpunten zijn niet gehaald.

Therapeutic value

Therapeutic value No judgement

This assessment does not indicate any potential inclusion in the package.

Expected patient volume per year

Patient volume 87 - 218

Market share is generally not included unless otherwise stated.

References Amyloidose Nederland
Additional remarks De geschatte incidentie ligt tussen de 5,1 en 12,8 per miljoen inwoners per jaar, dit betekent voor Nederland dus 87-218 patiënten per jaar.

Expected cost per patient per year

Potential total cost per year

Off label use

Off label use Unknown

Indication extension

Indication extensions Unknown

Other information