{"entity":{"id":"tagraxofusp","kind":"drug","name":"Tagraxofusp","aka":[],"tldr":"Tagraxofusp is a fusion of the growth factor IL-3 with a bacterial toxin that homes to CD123 on a rare, aggressive blood cancer.","summary":"Approved 2018 for blastic plasmacytoid dendritic cell neoplasm (BPDCN), the first CD123-directed therapy (CR/CRc 57% in untreated patients). Capillary leak syndrome is the boxed warning. Under study in CD123+ AML and CMML, usually in combination. Now joined in BPDCN by the CD123 ADC pivekimab sunirine (2026).","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Tagraxofusp","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Tagraxofusp"}],"tags":[],"related":[],"cancers":["aml"],"sections":[],"technologies":["monoclonal-antibody"],"targets":["cd123"],"drugs":[],"companies":["menarini"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06456463"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Elzonris","modality":"Recombinant cytotoxin (IL-3 fused to diphtheria toxin)","mechanism":"IL-3 domain binds CD123 (IL-3Rα); internalised diphtheria toxin fragment inhibits protein synthesis (ADP-ribosylation of EF-2).","approvals":[{"region":"US","year":2018,"indication":"Blastic plasmacytoid dendritic cell neoplasm, adults and children ≥2"}],"mechanismSteps":["IL-3 moiety binds CD123 on the blast","Receptor-mediated endocytosis internalises the fusion protein","Diphtheria toxin catalytic domain escapes to the cytosol","Elongation factor 2 is inactivated; protein synthesis stops; the cell dies"],"dosing":{"route":"IV over 15 minutes","schedule":"12 µg/kg daily on days 1-5 of a 21-day cycle; first cycle inpatient","monitoring":"Capillary leak syndrome (boxed warning): albumin, weight, oedema; hepatic enzymes","source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Elzonris"},"toxicity":[{"event":"Capillary leak syndrome","anyGradePct":55,"grade3PlusPct":9,"source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Elzonris","note":"Boxed warning"},{"event":"Hypoalbuminaemia","anyGradePct":51},{"event":"Transaminase elevation","anyGradePct":60}],"access":[],"regulatoryEvents":[]},"route":"/drugs/tagraxofusp/","neighbours":{"cancer":[{"id":"aml","kind":"cancer","name":"Acute myeloid leukaemia","route":"/cancers/aml/"},{"id":"bpdcn","kind":"cancer","name":"Blastic plasmacytoid dendritic cell neoplasm (BPDCN)","route":"/cancers/bpdcn/"}],"technology":[{"id":"monoclonal-antibody","kind":"technology","name":"Monoclonal antibodies","route":"/technologies/monoclonal-antibody/"}],"target":[{"id":"cd123","kind":"target","name":"CD123","route":"/targets/cd123/"}],"company":[{"id":"menarini","kind":"company","name":"Menarini / Stemline","route":"/companies/menarini/"}],"trial":[{"id":"nct06456463","kind":"trial","name":"A Study of Tagraxofusp in Combination With Venetoclax and Azacitidine in Adults With Untreated CD123+ Acute Myeloid Leukemia Who Cannot Undergo Intensive Chemotherapy","route":"/trials/nct06456463/"}]}}