Extended indication Confirmed infantile neuroaxonal dystrophy (INAD), homozygous for PLA2G6 deficiency in children aged
Therapeutic value No estimate possible yet
Registration phase Clinical trials

Product

Active substance Di-deuterated ethyl linoleate
Domain Neurological disorders
Reason of inclusion New medicine (specialité)
Main indication Muscular diseases other
Extended indication Confirmed infantile neuroaxonal dystrophy (INAD), homozygous for PLA2G6 deficiency in children aged 18 months to 10 years
Manufacturer Retrotope
Route of administration Oral
Therapeutical formulation Capsule
Budgetting framework Extramural (GVS)

Registration

Registration route Centralised (EMA)
ATMP No
Submission date 2023
Expected Registration 2024
Orphan drug Yes
Registration phase Clinical trials

Therapeutic value

Therapeutic value No estimate possible yet
Substantiation De studie heeft zijn primaire eindpunt niet gehaald maar zag wel verbetering in overleving.
References NCT03570931; https://www.fiercebiotech.com/biotech/retrotope-misses-main-endpoint-but-improves-survival-late-stage-trial-ultrarare-disorder

Expected patient volume per year

Patient volume

< 1

Market share is generally not included unless otherwise stated.

References SPS; erfelijkheid.nl; https://www.omroepbrabant.nl/nieuws/2051768/ouders-van-jasper-3-met-zeldzame-stofwisselingsziekte-vertellen-hun-emotionele-en-heftige-verhaal
Additional comments Wereldwijd zijn er 150 gevallen bekend, waarvan 1 in Nederland. De hoeveelheid patiënten in Nederland zal daarom erg beperkt zijn.

Expected cost per patient per year

Additional comments Een prijs is nog niet bekend maar gezien de hoge medical need en de beperkte hoeveelheid patiënten zal er, afhankelijk van de studieresultaten, een hoge prijs gevraagd kunnen worden.

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 Amyotrophic lateral sclerosis; Progressive supranuclear palsy
References Adis inisght

Other information

There is currently no futher information available.