Tagraxofusp

Tagraxofusp-erzs is a CD123-directed cytotoxin composed of recombinant human interleukin-3 (IL-3) and truncated diphtheria toxin (DT) fusion protein that inhibits protein synthesis and causes cell death in CD123-expressing cells.

• ELZONRIS is indicated for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adults and in pediatric patients 2 years and older.

• None.contraindicated

Premedicate with an H1-histamine antagonist, acetaminophen, corticosteroid and H2-histamine antagonist prior to each ELZONRIS infusion. ( 2.1 ) Administer ELZONRIS intravenously at 12 mcg/kg over 15 minutes once daily on days 1 to 5 of a 21-day cycle. ( 2.1 ) Administer the first cycle of ELZONRIS in the inpatient setting. Subsequent cycles may be administered in the inpatient or appropriate outpatient setting. ( 2.1 ) Additional important preparation and administration information is in full prescribing information. See full prescribing information for instructions on preparation and administration. ( 2.3 , 2.4 ) 2.1 Recommended Dosage Administer ELZONRIS at 12 mcg/kg intravenously over 15 minutes once daily on days 1 to 5 of a 21-day cycle. The dosing period may be extended for dose delays up to day 10 of the cycle. Continue treatment with ELZONRIS until disease progression or unacceptable toxicity. The dose is calculated based on the patient’s actual weight. Prior to the first dose of the first cycle, ensure serum albumin is greater than or equal to 3.2 g/dL before administering ELZONRIS. Premedicate patients with an H1-histamine antagonist (e.g., diphenhydramine hydrochloride), H2-histamine antagonist (e.g., famotidine), corticosteroid (e.g., 50 mg intravenous methylprednisolone or equivalent) and acetaminophen (or paracetamol) approximately 60 minutes prior to each ELZONRIS infusion. Administer Cycle 1 of ELZONRIS in the inpatient setting with patient observation through at least 24 hours after the last infusion.

Hypersensitivity: Monitor patients for signs/symptoms and treat appropriately. ( 5.2 ) Hepatotoxicity: Monitor ALT and AST. Interrupt ELZONRIS if the transaminases rise to greater than 5 times the upper limit of normal. ( 5.3 ) 5.1 Capillary Leak Syndrome Capillary leak syndrome (CLS), including life-threatening and fatal cases, has been reported among patients treated with ELZONRIS. In patients receiving ELZONRIS in clinical trials, the overall incidence of CLS was 53% (65/122), including Grade 1 or 2 in 43% (52/122) of patients, Grade 3 in 7% (8/122) of patients, Grade 4 in 1% (1/122) of patients, and four fatalities (3%) [see Adverse Reactions (6.1) ] . The median time to onset was 4 days (range - 1 to 46 days), and all but 5 patients experienced an event in Cycle 1. Before initiating therapy with ELZONRIS, ensure that the patient has adequate cardiac function and serum albumin is greater than or equal to 3.2 g/dL. During treatment with ELZONRIS, monitor serum albumin levels prior to the initiation of each dose of ELZONRIS and as indicated clinically thereafter, and assess patients for other signs or symptoms of CLS, including weight gain, new onset or worsening edema, including pulmonary edema, hypotension or hemodynamic instability [see Dosage and Administration (2.2) ]. 5.2 Hypersensitivity Reactions ELZONRIS can cause severe hypersensitivity reactions. In patients receiving ELZONRIS in clinical trials, hypersensitivity reactions were reported in 43% (53/122) of patients treated with ELZONRIS and were Grade ≥ 3 in 7% (9/122) [see Adverse Reactions (6.1) ] .

The following serious adverse drug reactions are described elsewhere in the labeling: Capillary Leak Syndrome [see Warnings and Precautions ( 5.1 ) ] Hypersensitivity Reactions [see Warnings and Precautions ( 5.2 ) ] Hepatotoxicity [see Warnings and Precautions ( 5.3 ) ] Most common adverse reactions (incidence ≥ 30%) are capillary leak syndrome, nausea, fatigue, pyrexia, peripheral edema, and weight increase. Most common laboratory abnormalities (incidence ≥ 50%) are decreases in albumin, platelets, hemoglobin, calcium, and sodium, and increases in glucose, ALT and AST. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Stemline Therapeutics, Inc. at 1-877-332-7961 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. Safety of ELZONRIS was assessed in a single-arm clinical trial that included 122 adults with newly diagnosed or relapsed/refractory myeloid malignancies, including 86 with BPDCN, treated with ELZONRIS 12 mcg/kg daily for 5 days of a 21-day cycle. The overall median number of cycles started was 2.5 (range, 1-76), and 4 in patients with BPDCN (range, 1-76). Four (3%) patients (4/122) had fatal adverse reactions, all of which were related to capillary leak syndrome.

Lactation: Advise women not to breastfeed ( 8.2 ) 8.1 Pregnancy Risk Summary Based on its mechanism of action, ELZONRIS has the potential for adverse effects on embryo-fetal development [see Clinical Pharmacology ( 12.1 )] . There are no available data on ELZONRIS use in pregnant women to inform a drug-associated risk of adverse developmental outcomes. Animal reproduction or developmental toxicity studies have not been conducted with tagraxofusp-erzs. Advise pregnant women of the potential risk to the fetus. The estimated background risk of major birth defects and miscarriage for the indicated population is unknown. All pregnancies have a risk of birth defect, loss, or other adverse outcomes. In the US general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4%, and 15% to 20%, respectively. 8.2 Lactation Risk Summary No data are available regarding the presence of ELZONRIS in human milk, the effects on the breastfed child, or the effects on milk production. Because of the potential for serious adverse reactions in breastfed children from ELZONRIS, breast feeding is not recommended during treatment and for 1 week after the last dose.

• Dec 21, 2018BLABLA761116Stemline Therapeutics Inc

1. 1Capillary Leak Syndrome12124%
2. 2Pyrexia448.9%
3. 3Death408.1%
4. 4Disease Progression357.1%
5. 5Off Label Use326.5%
6. 6Thrombocytopenia295.9%
7. 7Drug Ineffective275.5%
8. 8Tumour Lysis Syndrome255.1%
9. 9Weight Increased255.1%
10. 10Acute Kidney Injury234.7%
11. 11Blood Albumin Decreased234.7%
12. 12Transaminases Increased224.5%
13. 13Hypoalbuminaemia214.3%
14. 14Sepsis204.0%
15. 15Liver Function Test Increased193.8%

The 10 most recently updated of 22 ClinicalTrials.gov registrations naming Tagraxofusp as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• Combination of Tagraxofusp With Pacritinib in Patients With Intermediate-1 or Higher Myelofibrosis, Who Have Had Prior Therapy With the Approved JAK Inhibitors or in Which Therapy With the Approved JAK Inhibitors is Not Appropriate, Contraindicated or Declined
  Recruiting · Early phase 1 · Interventional · 20 enrolled · University of Kansas Medical Center
  NCT06414681updated 2026-05-06
• Tagraxofusp and Low-Intensity Chemotherapy for CD123-Positive Relapsed or Refractory AML
  Recruiting · Phase 1 · Phase 2 · Interventional · 20 enrolled · Stanford University
  NCT06561152updated 2026-04-14
• Venetoclax, SL-401, and Chemotherapy for the Treatment of Blastic Plasmacytoid Dendritic Cell Neoplasm
  Recruiting · Phase 2 · Interventional · 40 enrolled · M.D. Anderson Cancer Center
  NCT04216524updated 2026-04-14
• Phase I/II Study of Tagraxofusp in Combination With Decitabine for Patients With Myelomonocytic/Myeloproliferative Neoplasm and High Risk Myelodysplastic Syndromes
  Active not recruiting · Phase 1 · Phase 2 · Interventional · 64 enrolled · M.D. Anderson Cancer Center
  NCT05038592updated 2026-04-14
• Tagraxofusp and Azacitidine With Venetoclax in Newly Diagnosed Secondary AML After Hypomethylating Agents
  Recruiting · Phase 2 · Interventional · 53 enrolled · Joshua Zeidner
  NCT05442216updated 2026-04-08
• A Study of Tagraxofusp in Combination With Venetoclax and Azacitidine in Adults With Untreated CD123+ Acute Myeloid Leukemia Who Cannot Undergo Intensive Chemotherapy
  Recruiting · Phase 2 · Interventional · 83 enrolled · Stemline Therapeutics, Inc.
  NCT06456463updated 2026-03-09
• Tagraxofusp and Azacitidine for Maintenance Treatment in Patients With CD123 Positive AML and MDS Following Donor Hematopoietic Cell Transplant
  Recruiting · Phase 1 · Interventional · 43 enrolled · City of Hope Medical Center
  NCT06498973updated 2026-03-05
• A Multi-Site Break Through Cancer Trial: Targeting Measurable Residual Disease in Patients With Acute Myeloid Leukemia: A Phase 1/2 Study of Tagraxofusp, Azacitidine, and Venetoclax
  Recruiting · Phase 1 · Phase 2 · Interventional · 31 enrolled · Jacqueline Garcia, MD
  NCT07148180updated 2026-02-05
• Phase II Study Evaluating the Efficacy and Safety of the Combination of Tagraxofusp and Venetoclax in Treatment-naive Blastic Plasmacytoid Dendritic Cell Neoplasm Patients
  Not yet recruiting · Phase 2 · Interventional · 33 enrolled · French Innovative Leukemia Organisation
  NCT07007052updated 2025-11-26
• SL-401 in Combination With Azacitidine or Azacitidine/Venetoclax in Acute Myeloid Leukemia (AML), High-Risk Myelodysplastic Syndrome (MDS) or Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)
  Recruiting · Phase 1 · Interventional · 72 enrolled · Dana-Farber Cancer Institute
  NCT03113643updated 2025-11-04

Frequently asked questions

How does Tagraxofusp work?

Tagraxofusp-erzs is a CD123-directed cytotoxin composed of recombinant human interleukin-3 (IL-3) and truncated diphtheria toxin (DT) fusion protein that inhibits protein synthesis and causes cell death in CD123-expressing cells.

What is Tagraxofusp used for?

According to FDA labeling, Tagraxofusp carries indications including: ELZONRIS is indicated for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adults and in pediatric patients 2 years and older.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Tagraxofusp?

Tagraxofusp is classified as Other antineoplastic agents, CD123 Interaction, Cytotoxin, CD123 Interactions, Fusion Protein Interactions, Decreased Protein Synthesis, Increased Cellular Death.

What are the brand names for Tagraxofusp?

Tagraxofusp is marketed under brand names including Elzonris.

What are the contraindications for Tagraxofusp?

Tagraxofusp labeling lists contraindications including: None.. Always consult the full prescribing information and a clinician.