Arimoclomol Medicine 10 June 2020 Extended indication Amyotropic Lateral Sclerosis Therapeutic value Possible added value Total cost Registration phase No registration expected Product Active substance Arimoclomol Domain Neurological disorders Reason of inclusion New medicine (specialité) Main indication ALS Extended indication Amyotropic Lateral Sclerosis Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Orphazyme Portfolio holder Mechanism of action Other Route of administration Oral Therapeutical formulation Capsule Budgetting framework Extramural (GVS) Centre of expertise Additional remarks A hydroxylamine derivative thought to repair or induce destruction of misfolded proteins by amplifying molecular chaperone proteins and upregulating the cellular stress response. Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP Unknown Submission date Expected Registration Orphan drug Yes Registration phase No registration expected Reimbursement Medicine sluice Additional remarks Klinische studie heeft zijn primaire eindpunten niet gehaald. Therapeutic value Current treatment options Riluzole en in de toekomst mogelijk Ibudilast. Therapeutic value Possible added value This assessment does not indicate any potential inclusion in the package. 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 Frequency of administration Dosage per administration References NCT03491462 Additional remarks Expected patient volume per year Patient volume < 1,500 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References GIP databank, www.als.nl Additional remarks 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 Cost References Additional remarks Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Yes Indication extensions Sporadic Inclusion Body Myositis References clinicaltrials.gov Additional remarks fase 3 studie Other information Additional remarks