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Tagraxofusp

Elzonris

Extended indication Blastic plasmacytoid dendritic cell neoplasm (BPDCN)
Therapeutic value Possible added value
Registration phase Registration application pending

Product

Active substance Tagraxofusp
Domain Oncology and Hematology
Reason of inclusion New medicine (specialité)
Main indication Aggressive non-Hodgkin’s lymphoma
Extended indication Blastic plasmacytoid dendritic cell neoplasm (BPDCN)
Proprietary name Elzonris
Manufacturer Stemline
Mechanism of action Other
Route of administration Intravenous
Therapeutical formulation Intravenous drip
Budgetting framework Intermural (MSZ)
Additional remarks 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 Accelerated assessment
Submission date February 2019
Expected Registration October 2019
Orphan drug Yes
Registration phase Registration application pending

Therapeutic value

Current treatment options Daunorubicine en cytarabine
Therapeutic value Possible added value
Substantiation The efficacy of Elzonris was studied in two cohorts of patients in a single-arm clinical trial. The first trial cohort enrolled 13 patients with untreated BPDCN, and seven patients (54%) achieved complete remission (CR) or CR with a skin abnormality not indicative of active disease (CRc). The second cohort included 15 patients with relapsed or refractory BPDCN. One patient achieved CR and one patient achieved CRc.
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
Additional remarks BPDCN is moeilijke subgroep AML die het over het algemeen slecht doet op de standaard behandeling en ondanks (allogene) hematopoietische cel transplantatie vaak recidiveert.

Expected patient volume per year

Patient volume

3 - 10

Market share is generally not included unless otherwise stated.
References NKR
Additional remarks In 2015-2018 zijn er in totaal 23 diagnoses BPDCN geregistreerd, variërend tussen de 3 en 10 per jaar.

Expected cost per patient per year

References SmPC Vyxeos; Medicijnkosten.nl
Additional remarks Mogelijk hoge kosten. Voor de huidige behandeling met daunorubicine en cytarabine is de inschatting rond de €80.000 per patiënt per jaar.

Potential total cost per year

Additional remarks 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.