Treosulfan

Active substance
Treosulfan
Domain
Oncology and Hematology
Reason of inclusion in Horizonscan Geneesmiddelen
New medicine (specialité)
Main indication
Stem cell transplants
Extended indication
Treosulfan in combination with fludarabine is indicated as part of conditioning treatment prior to allogeneic haematopoietic stem cell transplantation (alloHSCT) in adult patients with malignant and non-malignant diseases, and in paediatric patients older than one month with malignant diseases.

1. Product

Proprietary name
Trecondi
Manufacturer
Medac
Mechanism of action
Alkylating agent
Route of administration
Intravenous
Therapeutical formulation
Intravenous drip
Budgetting framework
Extramural (GVS)
Additional comments
Alkylerend middel

2. Registration

Registration route
Centralised (EMA)
Type of trajectory
Normal trajectory
Submission date
February 2018
Expected Registration
June 2019
Orphan drug
Yes
Registration phase
Positive CHMP opinion
Additional comments
Positieve CHMP-opinie december 2018.

3. Therapeutic value

Current treatment options
Busulfan
Therapeutic value
No judgement
Duration of treatment
Average 3 day / days
Frequency of administration
1 times a day
Dosage per administration
10 g/m2
References
NCT00822393
Additional comments
Toedieningen op dagen -4, -3 en -2.

4. Expected patient volume per year

Patient volume

< 122

Market share is generally not included unless otherwise stated.

References
GIPdatabank
Additional comments
122 gebruikers van busulfan in 2015.

5. Expected cost per patient per year

Cost
5,000 - 6,000
References
Medicijnkosten.nl; Vademecum Hematologie
Additional comments
Busulfan volgens behandelschema vademecum hematologie voor een patient van 70 kg: 19 flacons, in totaal €5.883,70. Inmiddels is de prijs van Treosulfan bekend: injectieflacon peoder oplossing voor infusie van 1000MG FL: € 95,68 Uitgaande van gemiddeld 3 behandeldagen en een persoon van 1,8m2 kom je uit op € 5166,73 per patiënt per jaar.

6. Potential total cost per year

Total cost

671,000

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
Ovarian cancer, ewing sarcoma, ALL.
References
Clinicaltrials.gov

9. Other information

There is currently no futher information available.