Enasidenib Medicine 12 June 2018 Extended indication Acute myeloid leukaemia (AML), relapsed/refractory late-stage in patients with an IDH2 mutation. Therapeutic value No judgement Total cost € 3,500,000.00 Registration phase No registration expected Product Active substance Enasidenib Domain Oncology and Hematology Reason of inclusion New medicine (specialité) Main indication AML / MDS Extended indication Acute myeloid leukaemia (AML), relapsed/refractory late-stage in patients with an IDH2 mutation. Current proprietary name Already available biosimilars / generics Proprietary name Idhifa Manufacturer Celgene Portfolio holder Mechanism of action Enzyme inhibitor Route of administration Oral Therapeutical formulation Film-coated tablet Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks IDH2 inhibitor Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date July 2018 Expected Registration Orphan drug Yes Registration phase No registration expected Reimbursement Medicine sluice Additional remarks EUnetHTA had een beoordeling gepland voor deze indicatie (PTJA05). Registratie teruggetrokken in januari 2020 aangezien de fabrikant mogelijke risico's van het geneesmiddel aangekaart door de CHMP niet kan weerleggen. Therapeutic value Current treatment options Chemotherapie, stamceltransplantaties Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Momenteel te weinig informatie beschikbaar voor opname in de richtlijn. Duration of treatment Median 4.6 month / months Frequency of administration 1 times a day Dosage per administration 100 mg References Stein et al.Blood. 2017 Aug 10;130(6):722-731 Additional remarks fase 1-2 studie: Bij patiënten met relapsed/refractory AML, was de respons 40.3%, met een mediane respons van 5.8 maanden. Expected patient volume per year Patient volume < 35 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References NKR; Mycancergenome.org; kanker.nl Additional remarks 774 AML diagnoses in 2017. Volgens mycancergenome.org: 9.1% IDH2 mutations in AML. Kanker.nl: Bij ongeveer de helft van de patiënten die een complete remissie hebben bereikt, komt de ziekte na 1 tot 4 jaar toch weer terug. 774 x 0,091 x 0,5=35. 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 SPS Additional remarks Het geneesmiddel zal in de Verenigde Staten $24,872 per maand kosten (voor kortingen). Potential total cost per year Total cost € 3,500,000.00 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