Daratumumab Medicine 10 December 2019 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 Hematology Reason of inclusion Indication extension IND 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. Current proprietary name Already available biosimilars / generics Proprietary name Darzalex Manufacturer Janssen Portfolio holder Mechanism of action Immunostimulation Route of administration Subcutaneous Therapeutical formulation Solution for cutaneous use Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Antilichaam tegen CD38. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity New therapeutical formulation ATMP Unknown Submission date November 2020 Expected Registration June 2021 Orphan drug Yes Registration phase Registered Reimbursement Medicine sluice 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 This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration 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. Maximum patient volume for sluice 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 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. 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 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions 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. Additional remarks Other information Additional remarks