Ivosidenib

Active substance
Ivosidenib
Domain
Oncology and Hematology
Reason of inclusion in Horizonscan Geneesmiddelen
New medicine (specialité)
Main indication
AML / MDS
Extended indication
Acute myeloid leukaemia (AML), isocitrate dehydrogenase-1 (IDH1) mutation - first-line combination therapy.

1. Product

Proprietary name
TIBSOVO
Manufacturer
Agios
Mechanism of action
Enzyme inhibitor
Route of administration
Oral
Additional comments
Isocitrate dehydrogenase 1 inhibitor

2. Registration

Registration route
Centralised (EMA)
Expected Registration
2022
Orphan drug
No
Registration phase
Clinical trials

3. Therapeutic value

Current treatment options
Chemotherapie en haematopoietische stamcel transplantatie.
Therapeutic value
No judgement
Substantiation
De data is nog onbekend. De monotherapie in eerste lijn ziet er vooralsnog veelbelovend uit.
Duration of treatment
Not found
References
NCT02074839. Monotherapie eerste lijn. Combinatie eerste lijn nog niet gepubliceerd.

4. Expected patient volume per year

Patient volume

46 - 77

Market share is generally not included unless otherwise stated.

References
J Natl Cancer Inst. 2010 Jul 7; 102(13): 932–941. doi: 10.1093/jnci/djq187. Walter et al., Leukemia. 2015 Feb; 29(2): 312–320.; NKR
Additional comments
Er waren 774 AML diagnoses in 2017. Van de AML patiënten heeft 6 tot 10% heeft een IDH1mutatie. Dit betekent dat er mogelijk 46 tot 77 patiënten in Nederland in aanmerking zouden komen voor de behandeling.

5. Expected cost per patient per year

Cost
< 100,000
References
https://www.fiercepharma.com/pharma/agios-wins-fda-nod-for-targeted-aml-drug-tibsovo
Additional comments
Fiercepharma: "according to SunTrust analyst Yatin Suneja, Agios priced the drug at $26,115.00 a month". Dit zou neerkomen op zo'n €100.000 per patiënt per jaar uitgaande van een behandelduur van 4.1 maand. Gezien de kosten in de Verenigde Staten veelal wat hoger liggen dan in Europa is wel de verwachting dat het duur wordt maar minder dan €100.000.

6. Potential total cost per year

Total cost

6,150,000

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.