Daratumumab

Extended indication	Darzalex is indicated in combination with cyclophosphamide, bortezomib and dexamethasone for the treatment of adult patients with newly diagnosed systemic light chain (AL) amyloidosis.
Therapeutic value	No estimate possible yet
Total cost	16,091,360.00
Registration phase	Registered

Product

Active substance	Daratumumab
Domain	Oncology and Hematology
Reason of inclusion	Indication extension
Main indication	Multiple Myeloma
Extended indication	Darzalex is indicated in combination with cyclophosphamide, bortezomib and dexamethasone for the treatment of adult patients with newly diagnosed systemic light chain (AL) amyloidosis.
Proprietary name	Darzalex
Manufacturer	Janssen
Mechanism of action	Immunostimulation
Route of administration	Subcutaneous
Therapeutical formulation	Solution for cutaneous use
Budgetting framework	Intermural (MSZ)
Additional remarks	Antilichaam tegen CD38.

Registration

Registration route	Centralised (EMA)
Type of trajectory	Normal trajectory
Particularity	New therapeutical formulation
Submission date	November 2020
Expected Registration	June 2021
Orphan drug	Yes
Registration phase	Registered
Additional remarks	Positieve CHMP-opinie in mei 2021. Dit geneesmiddel is breed uitgesloten, wat betekent dat toekomstige indicaties automatisch in de sluis stromen.

Therapeutic value

Current treatment options	Bortezomib-cyclofosfamide-dexamethason of lenalidomide-dexamethason.
Therapeutic value	No estimate possible yet
Dosage per administration	16mg/kg
References	NCT03201965.
Additional remarks	Daratumumab zal wekelijks worden toegediend in de eerst 8 weken (2 cycli), daarna iedere 2 weken voor 4 cycli (cyclus 3-6) en daarna iedere 4 weken tot progressie of een maximum van 2 jaar. Jaar 1: 23 toedieningen; jaar 2: 13 toedieningen. Maximale behandelduur van 2 jaar.

Expected patient volume per year

Patient volume	< 160 Market share is generally not included unless otherwise stated.
References	De behandeling van AL-amyloïdose in Nederland anno 2013, HOVON;
Additional remarks	De incidentie wordt geschat op 8,9 per miljoen persoonsjaren; dat betekent dat er in Nederland 160 nieuwe patiënten per jaar zijn.

Patient volume

< 160

Market share is generally not included unless otherwise stated.

References

De behandeling van AL-amyloïdose in Nederland anno 2013, HOVON;

Additional remarks

De incidentie wordt geschat op 8,9 per miljoen persoonsjaren; dat betekent dat er in Nederland 160 nieuwe patiënten per jaar zijn.

Expected cost per patient per year

Cost	< 100,571.00
References	Fabrikant, G-standaard
Additional remarks	Jaar 1: €100.571; Jaar 2: €56.845. Op basis van lijstprijs. Maximale behandelduur van 2 jaar.

Potential total cost per year

Total cost	16,091,360.00 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.

Total cost

16,091,360.00

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

Indication extension	Yes
Indication extensions	In fase 3 klinische studies: Relapsed Refractory MM 1+ prior lines w/PomDex; Relapsed Refractory multiple myeloma w/carfilzomib/dex; Amyloidosis; Subcutaneous Formulation in patients with relapsed or refractory multiple myeloma; Smoldering multiple myeloma; Frontline multiple myeloma transplant ineligible in combination w/ bortezomib, lenalidomide and dexamethasone; Frontline multiple myeloma transplant eligible in combination w/ bortezomib, lenalidomide and dexamethasone.
References	Pipeline Janssen.

Other information

There is currently no futher information available.