Extended indication

Cholangiocarcinoma in patients with an isocitrate dehydrogenase 1 (IDH1) mutation, whose disease has

Therapeutic value

No estimate possible yet

Total cost

8,640,000.00

Registration phase

Clinical trials

Product

Active substance

Ivosidenib

Domain

Oncology and Hematology

Reason of inclusion

Indication extension

Main indication

Oncology other

Extended indication

Cholangiocarcinoma in patients with an isocitrate dehydrogenase 1 (IDH1) mutation, whose disease has progressed after one or two systemic therapies.

Proprietary name

Tibsovo

Manufacturer

Agios

Mechanism of action

Enzyme inhibitor

Route of administration

Oral

Budgetting framework

Intermural (MSZ)

Additional comments
First-in-class inhibitor of mutated IDH1 protein.

Registration

Registration route

Centralised (EMA)

Type of trajectory

Normal trajectory

Submission date

2022

Expected Registration

2022

Orphan drug

Yes

Registration phase

Clinical trials

Therapeutic value

Therapeutic value

No estimate possible yet

Frequency of administration

1 times a day

Dosage per administration

500 mg

References
NCT02989857.

Expected patient volume per year

Patient volume

< 32

Market share is generally not included unless otherwise stated.

References
NKR, Lowery et al. Lancet Gastroenterol Hepatol. 2019 Sep;4(9):711-720.
Additional comments
In 2017 waren er 86 patiënten met intra hepatisch cholangiocarcinoom waarvan ongeveer 25% verwacht wordt een mutatie te hebben in IDH1, dus dat zijn 22 patiënten. Daarnaast zijn er nog 70 diagnoses geweest met extra hepatisch cholangiocarcinoom waarvan ongeveer 14% verwacht wordt een mutatie te hebben in IDH1, dit betreft 10 patiënten. In totaal zijn dit 32 patiënten. Dit zijn allemaal patiënten van >18 jaar die gestart zijn met chemotherapie in de eerste lijn. Aangezien ivosidenib geïndiceerd is voor de tweede of derde lijn zal hiervan een deel in aanmerking komen voor deze behandeling.

Expected cost per patient per year

Cost

< 270,000.00

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 €270.000 per patiënt per jaar. 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 €270.000.

Potential total cost per year

Total cost

8,640,000.00

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
Clinicaltrials.gov.
Additional comments
Fase 3 studie.

Other information

There is currently no futher information available.