Extended indication Relapsed/Refractory systemic light chain (AL) amyloidosis
Therapeutic value No judgement
Total cost 1,450,800.00
Registration phase Withdrawn

Product

Active substance Ixazomib
Domain Oncology and Hematology
Reason of inclusion Indication extension
Main indication Multiple Myeloma
Extended indication Relapsed/Refractory systemic light chain (AL) amyloidosis
Proprietary name Ninlaro
Manufacturer Takeda
Mechanism of action Proteasome inhibitor
Route of administration Oral
Therapeutical formulation Capsule
Budgetting framework Intermural (MSZ)
Additional comments Zeer selectieve en reversibele proteasoomremmer

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
Orphan drug Yes
Registration phase Withdrawn
Additional comments 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
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

Expected patient volume per year

Patient volume

14 - 17

Market share is generally not included unless otherwise stated.

References De behandeling van AL-amyloïdose in Nederland anno 2013, HOVON
Additional comments 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
References G-standaard
Additional comments 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

1,450,800.00

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 No

Indication extension

Indication extension 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

Other information

There is currently no futher information available.