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Enasidenib

Idhifa

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.
Proprietary name Idhifa
Manufacturer celgene
Mechanism of action Enzyme inhibitor
Route of administration Oral
Therapeutical formulation Film-coated tablet
Budgetting framework Intermural (MSZ)
Additional remarks IDH2 inhibitor

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
Submission date July 2018
Orphan drug Yes
Registration phase No registration expected
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
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.
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
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

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.

Off label use

There is currently nothing known about off label use.

Indication extension

Indication extension Yes
Indication extensions Myelodysplastic syndromes
References adisinsight
Additional remarks fase 3 studie

Other information

There is currently no futher information available.