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Selinexor

Extended indication Patients with relapsed or refractory multiple myeloma who have received at least three prior lines o
Therapeutic value No judgement
Total cost 2,000,000.00
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 February 2021
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

Current treatment options Er zijn in de vierde lijn verschillende opties, vaak wordt voor een optie met lenalidomide gekozen.
Therapeutic value No judgement
Substantiation In patiënten die refractair zijn voor alle voorgaande behandeling (IMiD, PI, mAb) zal als laatste behandellijn selinexor kunnen worden overwogen indien patiënten in goede klinische conditie zijn. Een partiële respons is rond de 25% in die patiënten met een mediane duur van de respons van ruim 4 maanden. Het voordeel is dat het een andere class of drug is, er zijn met name gastro-intestinale bijwerkingen die in 25% ernstig zijn.
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; Long-term Outcomes in Patients With Multiple Myeloma. A Retrospective Analysis of the Dutch Population-based HAematological Registry for Observational Studies (PHAROS). Verelst, Silvia G.R.; HOVON MM.

Expected patient volume per year

Patient volume

< 50

Market share is generally not included unless otherwise stated.
Additional remarks Gezien de vele behandelopties komen naar schatting maximaal 50 patiënten per jaar hiervoor in aanmerking.

Expected cost per patient per year

Cost 40,000.00
References https://www.reuters.com/article/us-karyopharm-fda-idUSKCN1TY2FI.
Additional remarks In de Verenigde Staten 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

2,000,000.00

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.