Tagraxofusp

Active substance
Tagraxofusp
Domain
Oncology and Hematology
Reason of inclusion in Horizonscan Geneesmiddelen
New medicine (specialité)
Main indication
Aggressive non-Hodgkin's lymphoma
Extended indication
Blastic plasmacytoid dendritic cell neoplasm (BPDCN)

1. Product

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) .

2. 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

3. Therapeutic value

Current treatment options
Daunorubicine en cytarabine
Therapeutic value
Potential 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 comments
BPDCN is moeilijke subgroep AML die het over het algemeen slecht doet op de standaard behandeling en ondanks (allogene) hematopoietische cel transplantatie vaak recidiveert.

4. 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 en 10 per jaar.

5. Expected cost per patient per year

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

6. Potential total cost per year

Additional comments
Prijs nog niet bekend

7. Off label use

There is currently nothing known about off label use.

8. Indication extension

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

9. Other information

There is currently no futher information available.