Extended indication Elzonris is indicated as monotherapy for the first-line treatment of adult patients with blastic pla
Therapeutic value Possible added value
Total cost 650,000.00
Registration phase Registered

Product

Active substance Tagraxofusp
Domain Oncology and Hematology
Reason of inclusion New medicine (specialité)
Main indication Aggressive non-Hodgkin's lymphoma
Extended indication Elzonris is indicated as monotherapy for the first-line treatment of adult patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN). Elzonris should be administered under the supervision of a physician experienced in the use of anti-cancer agents.
Proprietary name Elzonris
Manufacturer Stemline
Mechanism of action Other
Route of administration Intravenous
Therapeutical formulation Intravenous drip
Budgetting framework Intermural (MSZ)
Additional comments Recombinant fusie-eiwit bestaande uit de eerste 388 aminozuren van diphtheria toxin (DT388) gefuseerd aan humaan interleukine-3 (IL-3).

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
Submission date February 2019
Expected Registration January 2021
Orphan drug Yes
Registration phase Registered
Additional comments Negatieve CHMP-opinie in juli 2020. In september 2020 heeft de fabrikant een verzoek tot herziening ingediend. Vervolgens is in november 2020 een positieve CHMP-opinie afgegeven.

Therapeutic value

Current treatment options Daunorubicine en cytarabine
Therapeutic value Possible added value
Substantiation BPDCN is moeilijke subgroep AML die het over het algemeen slecht doet op de standaard behandeling en ondanks (allogene) hematopoietische cel transplantatie vaak recidiveert.
Duration of treatment Average 5 day / days
Frequency of administration 1 times a day
Dosage per administration 1 dosis
References HOVON 103; NCT00397579; NCT02113982; FDA

Expected patient volume per year

Patient volume

3 - 10

Market share is generally not included unless otherwise stated.

References NKR
Additional comments In 2015-2018 zijn er in totaal 23 diagnoses BPDCN geregistreerd, variërend tussen de 3-10 per jaar.

Expected cost per patient per year

Cost 80,000.00 - 120,000.00
References SmPC Vyxeos; Medicijnkosten.nl; https://www.drugs.com/price-guide/elzonris.
Additional comments Mogelijk hoge kosten. Voor de huidige behandeling met daunorubicine en cytarabine is de inschatting rond de €80.000 per patiënt per jaar. De prijs voor Elzonris intraveneus oplossing (1.000mcg/mL) is ongeveer $26,662 (~€24.000) in de Verenigde Staten voor een hoeveelheid van 1 dosis. Dit zou bij 5 doses €120.000 bedragen.

Potential total cost per year

Total cost

650,000.00

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.

Additional comments Prijs nog niet bekend

Off label use

There is currently nothing known about off label use.

Indication extension

Indication extension Yes
Indication extensions Acute myeloid leukaemia; Multiple myeloma; Myelodysplastic syndromes; Myeloproliferative disorders.
References Clinicaltrials.gov.

Other information

There is currently no futher information available.