Ivosidenib Medicine 12 June 2019 Extended indication Acute myeloid leukaemia (AML), isocitrate dehydrogenase-1 (IDH1) mutation - first-line combination therapy. Therapeutic value No judgement Total cost € 16,200,000.00 Registration phase Registration application pending Product Active substance Ivosidenib Domain Oncology and Hematology Reason of inclusion New medicine (specialité) 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 First-in-class inhibitor of mutated IDH1 protein. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date January 2019 Expected Registration September 2019 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Goedgekeurd door de FDA, phase II Europa. Therapeutic value Current treatment options Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration 1 times a day Dosage per administration 500mg References NCT03173248 Additional remarks De mediane behandelduur uit voorgaande studies is 4.1 maanden (NCT02074839). FDA: De aanbevolen dosering ivosidenib is 500 mg oraal, eenmaal daags met of zonder voedsel of zonder voedsel tot progressie van de ziekte of onacceptabele toxiciteit. Voor patiënten zonder ziekte progressie of onacceptabele toxiciteit, is behandeling aanbevolen voor de duur van minimaal 6 maanden om een klinische respons te krijgen. Expected patient volume per year Patient volume 45 - 74 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. Cijfersoverkanker.nl Additional remarks Bij maximaal 8% van de AML patiënten zijn IDH1 mutaties gevonden in eerdere studies. Fiercepharma: "About 6% to 10% of AML patients have the IDH1 mutation, according to the biotech." Er zijn in totaal in 2017 774 patiënten met AML. Uitgaande van 6%-10% zou dat gaan om 45-75 patiënten. Expected cost per patient per year Cost € < 270,000.00 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 per month". Dit zou neerkomen op zo'n €270.000,00 p.p.p.j.. Gezien de kosten in Amerika veelal wat hoger liggen dan in Europa is wel de verwachting dat het duur wordt maar minder dan €270.000,00. Potential total cost per year Total cost € 16,200,000.00 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 Phase III Cholangiocarcinoma References Adisinsight en pipeline overview Agios Additional remarks Other information Additional remarks