Triheptanoin Medicine 08 June 2021 Extended indication Treatment of patients with Long chain fatty acid oxidation disorder (LC-FAOD). Therapeutic value No estimate possible yet Total cost Registration phase Clinical trials Product Active substance Triheptanoin Domain Metabolism and Endocrinology Reason of inclusion New medicine (specialité) Main indication Metabolic diseases Extended indication Treatment of patients with Long chain fatty acid oxidation disorder (LC-FAOD). Current proprietary name Already available biosimilars / generics Proprietary name Dojolvi Manufacturer Ultragenyx Portfolio holder Mechanism of action Other Route of administration Oral Therapeutical formulation Liquid Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Synthetische triglyceride die kan worden gebruikt als extra energiebron in de hersenen. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date September 2022 Expected Registration September 2023 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Wordt waarschijnlijk in de tweede helft van 2022 ingediend bij de EMA. Therapeutic value Current treatment options Ketogeen dieet Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Werkt met name bij de epileptische aanvallen, niet bij de bewegingsstoornissen. Als het werkt is er zeker meerwaarde. Duration of treatment Frequency of administration Dosage per administration References NCT02201368 Additional remarks Expected patient volume per year Patient volume < 114 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References SPS Additional remarks De prevalentie van LC-FAOD wordt geschat op 0,67 per 100.000 inwoners in westerse landen. Dit zou voor Nederland betekenen dat er maximaal 114 patiënten voor Triheptanoin in aanmerking zouden komen. Expected cost per patient per year Cost References Additional remarks In de Verenigde Staten ligt de prijs voor per patiënt per jaar op $138.000. De prijs in Nederland is nog niet bekend. 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 References Additional remarks Other information Additional remarks