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 comments
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 comments
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 comments
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 comments
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 comments
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 comments
fase 3 studie

Other information

There is currently no futher information available.