Ivosidenib Medicine 10 December 2019 Extended indication Cholangiocarcinoma in patients with an isocitrate dehydrogenase 1 (IDH1) mutation, whose disease has progressed after one or two systemic therapies Therapeutic value No judgement Total cost € 8,640,000.00 Registration phase Clinical trials Product Active substance Ivosidenib Domain Oncology and Hematology Reason of inclusion Indication extension IND Main indication Oncology other Extended indication Cholangiocarcinoma in patients with an isocitrate dehydrogenase 1 (IDH1) mutation, whose disease has progressed after one or two systemic therapies 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 2020 Expected Registration 2021 Orphan drug Unknown Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks 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 500 mg References NCT02989857 Additional remarks Expected patient volume per year Patient volume < 32 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References NKR, Lowery et al. Lancet Gastroenterol Hepatol. 2019 Sep;4(9):711-720 Additional remarks In 2017 waren er 86 patiënten met intrahepatisch cholangiocarcinoom waarvan ongeveer 25% verwacht wordt een mutatie te hebben in IDH1, dus dat zijn 22 patiënten. Daarnaast zijn er nog 70 diagnoses geweest met extrahepatisch cholangiocarcinoom waarvan ongeveer 14% verwacht wordt een mutatie te hebben in IDH1, dit betreft 10 patiënten. In totaal zijn dit 32 patiënten. Dit zijn allemaal patiënten van >18 jaar die gestart zijn met chemotherapie in de eerste lijn. Aangezien ivosidenib geïndiceerd is voor de 2e of 3e lijn zal hiervan een deel in aanmerking komen voor deze behandeling. 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 a month". Dit zou neerkomen op zo'n €270.000 per patiënt per jaar. 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 €270.000. Potential total cost per year Total cost € 8,640,000.00 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 clinicaltrials.gov Additional remarks Fase 3 studie Other information Additional remarks