Di-deuterated ethyl linoleate Medicine 07 June 2022 Extended indication Confirmed infantile neuroaxonal dystrophy (INAD), homozygous for PLA2G6 deficiency in children aged 18 months to 10 years Therapeutic value No estimate possible yet Total cost 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 Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Retrotope Portfolio holder Mechanism of action Unknown Route of administration Oral Therapeutical formulation Capsule Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP No Submission date 2023 Expected Registration 2024 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation De studie heeft zijn primaire eindpunt niet gehaald maar zag wel verbetering in overleving. Duration of treatment Frequency of administration Dosage per administration References NCT03570931; https://www.fiercebiotech.com/biotech/retrotope-misses-main-endpoint-but-improves-survival-late-stage-trial-ultrarare-disorder Additional remarks Expected patient volume per year Patient volume < 1 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References SPS; erfelijkheid.nl; https://www.omroepbrabant.nl/nieuws/2051768/ouders-van-jasper-3-met-zeldzame-stofwisselingsziekte-vertellen-hun-emotionele-en-heftige-verhaal Additional remarks 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 Cost References Additional remarks 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 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 Amyotrophic lateral sclerosis; Progressive supranuclear palsy References Adis inisght Additional remarks Other information Additional remarks