Cytarabine / daunorubicin (liposomaal)

Active substance
Cytarabine / daunorubicin (liposomaal)
Domain
Oncology and Hematology
Reason of inclusion in Horizonscan Geneesmiddelen
New medicine (specialité)
Main indication
AML / MDS
Extended indication
Vyxeos is indicated for the treatment of adults with newly diagnosed, therapy-related acute myeloid leukaemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC).

1. Product

Proprietary name
Vyxeos
Manufacturer
Jazz
Mechanism of action
Combination therapy
Route of administration
Intravenous
Therapeutical formulation
Intravenous drip
Budgetting framework
Intermural (MSZ)

2. Registration

Registration route
Centralised (EMA)
Type of trajectory
Accelerated assessment
Submission date
December 2017
Expected Registration
August 2018
Orphan drug
Yes
Registration phase
Registered and reimbursed
Additional comments
Positieve CHMP-opinie in juli 2018. Geregistreerd in augustus 2018.

3. 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).
Therapeutic value
Potential added value
Substantiation
EMA geeft aan dat er mogelijke meerwaarde is. Expertopinie: mogelijkheid tot hoger doseren met minder toxiciteit.
Duration of treatment
Average 11 day / days
Frequency of administration
1 times every 2 days
Dosage per administration
100 /44 mg/m2
References
SmPC Vyxeos

4. Expected patient volume per year

Patient volume

< 147

Market share is generally not included unless otherwise stated.

References
NKR 2015
Additional comments
110 diagnoses AML met myelodysplasie-gerelateerde veranderingen; 37 diagnoses AML na eerdere cytotoxische chemo- en/of radiotherapie.

5. Expected cost per patient per year

Cost
80,000
References
SmPC Vyxeos; Medicijnkosten.nl
Additional comments
Op basis van de SmPC worden er 14 flacons verwacht per behandeling. Per stuk kosten deze flacons €5.668. In totaal komt dit dus neer op €79.352.

6. Potential total cost per year

Total cost

11,760,000

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
No
References
SPS

9. Other information

There is currently no futher information available.