Extended indication Extension of indication to add treatment of relapsed/refractory AML in paediatric patients
Therapeutic value Possible added value
Total cost 595,140.00
Registration phase No registration expected

Product

Active substance Cytarabine / daunorubicin (liposomaal)
Domain Hematology
Reason of inclusion Indication extension
Main indication AML / MDS
Extended indication Extension of indication to add treatment of relapsed/refractory AML in paediatric patients
Proprietary name Tav Vyxeos
Manufacturer Jazz
Mechanism of action Combination therapy
Route of administration Intravenous
Therapeutical formulation Intravenous drip
Budgetting framework Intermural (MSZ)

Registration

Registration route Centralised (EMA)
Submission date December 2021
Orphan drug Yes
Registration phase No registration expected
Additional comments CHMP Maart 2022:"Concluding a review of an application to extend the use of Vyxeos liposomal* (daunorubicin / cytarabine), the CHMP did not recommend including the treatment of young patients (aged 1 to 21 years) with acute myeloid leukaemia (AML) to the authorised indications. The Committee agreed that relevant data from the study submitted with the application should be included in the medicine’s product information so that healthcare professionals have access to up-to-date data on the effects of Vyxeos liposomal in patients with relapsing or refractory AML."

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 Possible added value
Substantiation EMA geeft aan dat er mogelijke meerwaarde is. De meerwaarde is gebaseerd op het artikel van Cooper alhoewel de resultaten nog beperkt zijn qua follow up. Het is ook duidelijk dat een liposomaal product is minder cardiotoxisch is en dat is van belang nu daunoxome niet meer te krijgen is.
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

Expected patient volume per year

Patient volume

5 - 10

Market share is generally not included unless otherwise stated.

References Expertopinie
Additional comments Op basis van een expertopinie wordt er ingeschat dat er ongeveer tussen de 5-10 patiënten per jaar met een recidief/refractaire AML bij kinderen in aanmerking zullen komen.

Expected cost per patient per year

Cost > 79,352.00
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.

Potential total cost per year

Total cost

595,140.00

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

There is currently nothing known about indication extensions.

Other information

There is currently no futher information available.