Extended indication Acute myeloid leukaemia (AML), relapsed/refractory late-stage in patients with an IDH2 mutation.
Therapeutic value No judgement
Total cost 3,300,000.00
Registration phase Registration application pending

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 Conditional marketing authorisation
Submission date July 2018
Expected Registration September 2019
Orphan drug Yes
Registration phase Registration application pending

Therapeutic value

Current treatment options EMA: Treatment for AML is complex and depends on a number of factors including the extent of the disease, whether it has been treated before, and the patient’s age, symptoms and general state of health. At the time of designation, the main treatments for AML were chemotherapy (medicines to treat cancer) and haematopoietic (blood) stem-cell transplantation (a complex procedure where the patient receives stem cells from a matched donor to help restore the bone marrow). Fabrikant: HiDAC, LDAC
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 patienten met relapsed/refractory AML, was de respons 40.3%, met een mediane respons van 5.8 maanden.

Expected patient volume per year

Patient volume

< 33

Market share is generally not included unless otherwise stated.

References NKR2015; Mycancergenome.org; kanker.nl
Additional remarks 714 AML diagnoses. 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. 714 x 0,091 x 0,5=33

Expected cost per patient per year

Cost 100,000.00
References SPS
Additional remarks Het geneesmiddel zal in de VS $24,872 per maand kosten (voor kortingen).

Potential total cost per year

Total cost

3,300,000.00

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 No
References Fabrikant
Additional remarks Er lopen wel meerdere studies voor andere aandoeningen maar nog niet in phase III.

Other information

There is currently no futher information available.