Plerixafor

Active substance
Plerixafor
Domain
Oncology and Hematology
Reason of inclusion in Horizonscan Geneesmiddelen
Indication extension
Main indication
Stem cell transplants
Extended indication
Extension of Indication to include paediatric patients aged 1 to 18 years. Mozobil is indicated in combination with G-CSF to enhance mobilisation of haematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in children with lymphoma or solid malignant tumours, either: • pre-emptively, when circulating stem cell count on the predicted day of collection after adequate mobilization with G-CSF (with or without chemotherapy) is expected to be insufficient with regards to desired hematopoietic stem cells yield, or • who previously failed to collect sufficient haematopoietic stem cells.

1. Product

Proprietary name
Mozobil
Manufacturer
Genzyme
Mechanism of action
Receptor antagonist
Route of administration
Subcutaneous
Therapeutical formulation
Injection
Budgetting framework
Intermural (MSZ)
Additional comments
Alpha chemokine receptor CXCR4 antagonist.

2. Registration

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

3. Therapeutic value

Current treatment options
Oogsten met GCSF alleen
Therapeutic value
No judgement
Duration of treatment
Not found
Frequency of administration
1 times a day
Dosage per administration
0,24 mg/kg
References
EPAR
Additional comments
Plerixafor dient 6 tot 11 uur vóór aanvang van elke aferese te worden toegediend door middel van een subcutane injectie, na een voorbehandeling van 4 dagen met G-CSF. Bij klinische onderzoeken werd plerixafor meestal gedurende 2 tot 4 (en tot maximaal 7) opeenvolgende dagen gebruikt.

4. Expected patient volume per year

Patient volume

6 - 10

Market share is generally not included unless otherwise stated.

References
GIPdatabank; CBS
Additional comments
In 2015 werden 11 volwassenen behandeld met plerixafor. De verhouding van 1-18 jarigen ten opzichte van 18+ in Nederland in 2017 was 1:3,7. Dit zou betekenen dat er 3 kinderen in aanmerking zouden komen voor deze indicatie-uitbreiding. In het prinses Maxima centrum werden in 2018 6 kinderen behandeld met Mozobil, in 2019 tot op heden 4 kinderen.

5. Expected cost per patient per year

Cost
< 11,000
References
GIPdatabank; EPAR
Additional comments
In 2015 werd er €10.648 vergoed per gebruiker van plerixafor. Aangezien plerixafor per kg lichaamsgewicht wordt toegediend zullen de kosten per patiënt per jaar wellicht iets lager uitvallen.

6. Potential total cost per year

Total cost

88,000

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

Off label use
Yes
Indications off label use
Eventueel bij autologe SCT bij hersentumoren

8. Indication extension

Indication extension
No

9. Other information

There is currently no futher information available.