Active substance Arimoclomol
Domain Neurological disorders
Reason of inclusion New medicine (specialité)
Main indication ALS
Extended indication Amyotropic Lateral Sclerosis


Manufacturer Orphazyme
Mechanism of action Other
Route of administration Oral
Therapeutical formulation Capsule
Budgetting framework Extramural (GVS)
Additional comments A hydroxylamine derivative thought to repair or induce destruction of misfolded proteins by amplifying molecular chaperone proteins and upregulating the cellular stress response.


Registration route Centralised (EMA)
Submission date 2021
Expected Registration 2022
Orphan drug Yes
Registration phase Clinical trials

Therapeutic value

Current treatment options Riluzole en in de toekomst mogelijk Ibudilast.
Therapeutic value Potential added value
Substantiation Arimoclomol is mogelijk alleen effectief wanneer er sprake is van een mutatie in het SOD1-gen. De huidige fase 3 studie includeert alle ALS patiënten. Indien de resultaten positief zijn, komt het voor alle ALS patiënten beschikbaar. De resultaten van de fase 3 studie worden in december 2020 verwacht.
Duration of treatment Not found
References NCT03491462

Expected patient volume per year

Patient volume

< 1,500

Market share is generally not included unless otherwise stated.

References GIP databank,
Additional comments In Nederland leven gemiddeld 1.500 mensen met ALS. (In Nederland in 2016 betrof het aantal riluzole gebruikers: 1.071 patiënten). Indien de resultaten van de fase 3 studie positief blijken te zijn, komt dit geneesmiddel op de markt voor alle ALS patiënten.

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

Indication extension Yes
Indication extensions Sporadic Inclusion Body Myositis
Additional comments fase 3 studie

Other information

There is currently no futher information available.