Enasidenib

Active substance
Enasidenib
Domain
Oncology and Hematology
Reason of inclusion in Horizonscan Geneesmiddelen
New medicine (specialité)
Main indication
AML / MDS
Extended indication
Acute myeloid leukaemia (AML), relapsed/refractory late-stage in patients with an IDH2 mutation.

1. Product

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

2. 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.

3. 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.

4. 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.

5. Expected cost per patient per year

Cost
100,000
References
SPS
Additional comments
Het geneesmiddel zal in de Verenigde Staten $24,872 per maand kosten (voor kortingen).

6. Potential total cost per year

Total cost

3,500,000

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.

7. Off label use

There is currently nothing known about off label use.

8. Indication extension

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

9. Other information

There is currently no futher information available.