Extended indication Extension of Indication to include paediatric patients aged 1 to 18 years. Mozobil is indicated in c
Therapeutic value No judgement
Total cost 88,000.00
Registration phase Positive CHMP opinion

Product

Active substance Plerixafor
Domain Oncology and Hematology
Reason of inclusion 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.
Proprietary name Mozobil
Manufacturer Genzyme
Mechanism of action Receptor antagonist
Route of administration Subcutaneous
Therapeutical formulation Injection
Budgetting framework Intermural (MSZ)
Additional remarks Alpha chemokine receptor CXCR4 antagonist.

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 remarks Positieve CHMP-opinie maart 2019.

Therapeutic value

Current treatment options Oogsten met GCSF alleen
Therapeutic value No judgement
Frequency of administration 1 times a day
Dosage per administration 0,24 mg/kg
References EPAR
Additional remarks 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.

Expected patient volume per year

Patient volume

6 - 10

Market share is generally not included unless otherwise stated.

References GIPdatabank; CBS
Additional remarks 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.

Expected cost per patient per year

Cost < 11,000.00
References GIPdatabank; EPAR
Additional remarks 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.

Potential total cost per year

Total cost

88,000.00

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

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

Indication extension

Indication extension No

Other information

There is currently no futher information available.