Extended indication Patients with relapsed or refractory multiple myeloma who have received at least three prior lines o
Therapeutic value No judgement
Total cost 6,640,000.00
Registration phase Registration application pending

Product

Active substance Selinexor
Domain Oncology and Hematology
Reason of inclusion New medicine (specialité)
Main indication Multiple Myeloma
Extended indication Patients with relapsed or refractory multiple myeloma who have received at least three prior lines of therapy and whose disease is refractory to at least one PI, one IMiD, and one anti-CD38 monoclonal antibody, and to their most recent treatment regimen.
Manufacturer Karyopharm
Mechanism of action Other
Route of administration Oral
Budgetting framework Intermural (MSZ)
Additional remarks CRM1 nuclear export inhibitor

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
Submission date February 2019
Expected Registration March 2020
Orphan drug Yes
Registration phase Registration application pending
Additional remarks Werd eerder beoordeeld via accelerated assessment, maar dat is gewijzigd per juni 2019.

Therapeutic value

Therapeutic value No judgement
Duration of treatment Median 9 week / weeks
Frequency of administration 2 times a week
Dosage per administration 80 mg
References NCT02336815; Chari et al. N Engl J Med 2019; 381:727-738

Expected patient volume per year

Patient volume

< 166

Market share is generally not included unless otherwise stated.

References Pakketadvies daratumumab; Yong et al. Br J Haematol. 2016 Oct;175(2):252-264; Willenbacher et al. PLoS One. 2016 Mar 3;11(3):e0147381
Additional remarks Uit het pakketadvies voor daratumumab blijkt dat 830 patiënten in de eerste lijn behandeld worden. Het geschatte aantal patiënten wat in de derde lijn behandeld wordt voor multiple myeloom is 418. Van het totaal van 830 patiënten zal op basis van de literatuur ongeveer 15-20% in de 4e lijn behandeld, dit zou dus neerkomen op maximaal 166 patiënten. De inschatting is dat dit aantal nog lager zal liggen gegeven de vele behandelopties.

Expected cost per patient per year

Cost 40,000.00
References https://www.reuters.com/article/us-karyopharm-fda-idUSKCN1TY2FI
Additional remarks In de VS is de lijstprijs per maand bepaald op $22.000 (~€20.000). De mediane behandelduur is 9 weken wat neerkomt op ongeveer €40.000.

Potential total cost per year

Total cost

6,640,000.00

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

There is currently nothing known about off label use.

Indication extension

Indication extension Yes
Indication extensions Liposarcoma; Endometrial cancer; Acute myeloid leukaemia; Breast cancer; Cervical cancer; Cutaneous T-cell lymphoma; Diffuse large B cell lymphoma; Glioblastoma; Myelodysplastic syndromes; Neuroendocrine tumours; Ovarian cancer; Peripheral T-cell lymphoma; Prostate cancer; Small cell lung cancer; Squamous cell cancer; Thymoma; Chronic myelomonocytic leukaemia; Non-Hodgkin's lymphoma; Non-small cell lung cancer; Soft tissue sarcoma
References Clinicaltrials.gov

Other information

There is currently no futher information available.