Tagraxofusp Medicine 12 June 2019 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. Therapeutic value Possible added value Total cost € 700,000.00 Registration phase Registered Product Active substance Tagraxofusp Domain Oncology Reason of inclusion New medicine (specialité) Main indication Unknown 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. Current proprietary name Already available biosimilars / generics Proprietary name Elzonris Manufacturer Stemline Portfolio holder Mechanism of action Other Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise 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 Normal trajectory Particularity Unknown ATMP Unknown Submission date February 2019 Expected Registration January 2021 Orphan drug Yes Registration phase Registered Reimbursement Medicine sluice Additional remarks 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. De fabrikant geeft aan dat er zo spoedig mogelijk een vergoedingsdossier wordt opgestart. Therapeutic value Current treatment options Daunorubicine en cytarabine Therapeutic value Possible added value This assessment does not indicate any potential inclusion in the package. Substantiation BPDCN is moeilijke subgroep AML die het over het algemeen slecht doet op de standaard behandeling en ondanks (allogene) hematopoiëtische 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 Additional remarks Expected patient volume per year Patient volume 3 - 10 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice 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 Cost € 80,000.00 - 120,000.00 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. References SmPC Vyxeos; Medicijnkosten.nl; https://www.drugs.com/price-guide/elzonris. Additional remarks 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 (ongeveer €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 € 700,000.00 Total cost for sluice Additional remarks Prijs nog niet bekend Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Yes Indication extensions Acute myeloid leukaemia; Multiple myeloma; Myelodysplastic syndromes; Myeloproliferative disorders. References Clinicaltrials.gov. Additional remarks Other information Additional remarks