Ivosidenib Medicine 10 June 2020 Extended indication Acute myeloid leukaemia (AML), isocitrate dehydrogenase-1 (IDH1) mutation - first-line combination therapy. Therapeutic value No judgement Total cost € 6,200,000.00 Registration phase No registration expected Product Active substance Ivosidenib Domain Oncology and Hematology Reason of inclusion Indication extension IND Main indication AML / MDS Extended indication Acute myeloid leukaemia (AML), isocitrate dehydrogenase-1 (IDH1) mutation - first-line combination therapy. Current proprietary name Already available biosimilars / generics Proprietary name TIBSOVO Manufacturer Agios Portfolio holder Mechanism of action Enzyme inhibitor Route of administration Oral Therapeutical formulation Unknown Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Isocitrate dehydrogenase 1 inhibitor Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP Unknown Submission date Expected Registration Orphan drug No Registration phase No registration expected Reimbursement Medicine sluice Additional remarks De fabrikant geeft aan dat de registratieaanvraag teruggetrokken is. Er wordt verwacht dat de data niet afdoende is om een positieve-CHMP opinie te ontvangen. Therapeutic value Current treatment options Chemotherapie en haematopoietische stamcel transplantatie. Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation De data is nog onbekend. De monotherapie in eerste lijn ziet er vooralsnog veelbelovend uit. Duration of treatment Frequency of administration Dosage per administration References NCT02074839. Monotherapie eerste lijn. Combinatie eerste lijn nog niet gepubliceerd. Additional remarks Expected patient volume per year Patient volume 46 - 77 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References J Natl Cancer Inst. 2010 Jul 7; 102(13): 932–941. doi: 10.1093/jnci/djq187. Walter et al., Leukemia. 2015 Feb; 29(2): 312–320.; NKR Additional remarks Er waren 774 AML diagnoses in 2017. Van de AML patiënten heeft 6%-10% een IDH1mutatie. Dit betekent dat er mogelijk 46 tot 77 patiënten in Nederland in aanmerking zouden komen voor de behandeling. Expected cost per patient per year Cost € < 100,000.00 This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year. References https://www.fiercepharma.com/pharma/agios-wins-fda-nod-for-targeted-aml-drug-tibsovo Additional remarks Fiercepharma: "according to SunTrust analyst Yatin Suneja, Agios priced the drug at $26,115.00 a month". Dit zou neerkomen op zo'n €100.000 per patiënt per jaar uitgaande van een behandelduur van 4.1 maand. Gezien de kosten in de Verenigde Staten veelal wat hoger liggen dan in Europa is wel de verwachting dat het duur wordt maar minder dan €100.000. Potential total cost per year Total cost € 6,200,000.00 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 Myelodysplastic syndromes References adisinsight Additional remarks Fase 3 studie Other information Additional remarks