Arimoclomol

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

1. Product

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.

2. Registration

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

3. 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

4. Expected patient volume per year

Patient volume

< 1,500

Market share is generally not included unless otherwise stated.

References
GIP databank, www.als.nl
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.

5. Expected cost per patient per year

There is currently nothing known about the expected cost.

6. Potential total cost per year

There is currently nothing known about the possible total cost.

7. Off label use

There is currently nothing known about off label use.

8. Indication extension

Indication extension
Yes
Indication extensions
Sporadic Inclusion Body Myositis
References
clinicaltrials.gov
Additional comments
fase 3 studie

9. Other information

There is currently no futher information available.