Ixazomib Medicine 12 June 2019 Extended indication Relapsed/Refractory systemic light chain (AL) amyloidosis Therapeutic value No judgement Total cost € 1,497,600.00 Registration phase Withdrawn Product Active substance Ixazomib Domain Oncology and Hematology Reason of inclusion Indication extension IND Main indication Multiple Myeloma Extended indication Relapsed/Refractory systemic light chain (AL) amyloidosis Current proprietary name Already available biosimilars / generics Proprietary name Ninlaro Manufacturer Takeda Portfolio holder Mechanism of action Proteasome inhibitor Route of administration Oral Therapeutical formulation Capsule Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Zeer selectieve en reversibele proteasoomremmer Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date Expected Registration Orphan drug Yes Registration phase Withdrawn Reimbursement Medicine sluice Additional remarks De TOURMALINE-AL1 heeft de primaire eindpunten niet gehaald en is door Takeda stopgezet. Therapeutic value Current treatment options Bortezomib-cyclofosfamide-dexamethason of lenalidomide-dexamethason. Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation De TOURMALINE-AL1 heeft de primaire eindpunten niet gehaald en is door Takeda stopgezet. Duration of treatment Median 5 week / weeks Frequency of administration 3 times every 4 weeks Dosage per administration 4 mg References NCT01659658 Additional remarks Expected patient volume per year Patient volume 14 - 17 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 persoonjaren; dat betekent dat er in Nederland 160 nieuwe patiënten per jaar zijn. De inschatting is dat ongeveer 35% relpase/refractory is (56). De fabrikant verwacht een marktaandeel van 25-30% (14-17). Expected cost per patient per year Cost € 93,600.00 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 G-standaard Additional remarks Kosten per patiënt per jaar zijn berekend op basis van AIP en gelden voor patiënten die een volledig jaar behandeld worden. Momenteel is ixazomib vergoed middels een financiële afspraak tussen Takeda en de zorgverzekeraars. Potential total cost per year Total cost € 1,497,600.00 Total cost for sluice Additional remarks Off label use Off label use No Indications off label use References Additional remarks Indication extension Indication extensions Yes Indication extensions - Maintenance therapy for multiple myeloma nontransplant (NSCT) patients - Newly diagnosed multiple myeloma patients ineligible for transplantation and with elevated risk cytogenetics - 1L Post-ASCT maintenance References ASH 2018 Additional remarks Other information Additional remarks