Tagraxofusp Medicine 12 June 2019 Extended indication Blastic plasmacytoid dendritic cell neoplasm (BPDCN) Therapeutic value Possible added value Total cost € 700,000.00 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) 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 March 2020 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks 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) 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 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 (1.000 mcg/mL) is ongeveer $26,662 (~€24.000) in de VS 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