Naxitamab

Naxitamab-gqgk binds to the glycolipid GD2. GD2 is a disialoganglioside that is overexpressed on neuroblastoma cells and other cells of neuroectodermal origin, including the central nervous system and peripheral nerves.

• DANYELZA is indicated, in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF), for the treatment of pediatric patients 1 year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy. This indication is approved under accelerated approval based on overall response rate and duration of response [see Clinical Studies (14) ] .

• DANYELZA is contraindicated in patients with a history of severe hypersensitivity reaction to naxitamab-gqgk. Reactions have included anaphylaxis [see Warnings and Precautions (5.1) ].contraindicated

The recommended dosage of DANYELZA is 3 mg/kg/day (up to 150 mg/day), administered as an intravenous infusion after dilution on Days 1, 3, and 5 of each treatment cycle. Treatment cycles are repeated every 4 weeks until complete response or partial response, followed by 5 additional cycles every 4 weeks. Subsequent cycles may be repeated every 8 weeks. Discontinue DANYELZA and GM-CSF for disease progression or unacceptable toxicity. Administer GM-CSF subcutaneously prior to and during each treatment cycle as recommended. ( 2.1 ) 2.1 Recommended Dosage The recommended dosage of DANYELZA is 3 mg/kg/day (up to 150 mg/day) on Days 1, 3, and 5 of each treatment cycle, administered as an intravenous infusion after dilution [see Dosage and Administration (2.4 and 2.5) ] in combination with GM-CSF subcutaneously as shown in Table 1. Refer to the GM-CSF Prescribing Information for recommended dosing information. Treatment cycles are repeated every 4 weeks until complete response or partial response, followed by 5 additional cycles every 4 weeks. Subsequent cycles may be repeated every 8 weeks. Discontinue DANYELZA and GM-CSF for disease progression or unacceptable toxicity. Administer pre-infusion medications and supportive treatment, as appropriate, during infusion. [see Dosage and Administration (2.2) ] The recommended dosage regimen for each treatment cycle is described below and in Table 1: Days -4 to 0: administer GM-CSF 250 µg/m 2 /day by subcutaneous injection, beginning 5 days prior to DANYELZA infusion.

Neurotoxicity: Peripheral neuropathy, neurological disorders of the eye, and prolonged urinary retention have also occurred. Permanently discontinue as recommended. ( 2.3 , 5.2 ) Myocarditis: Withhold, reduce dose, or discontinue based on severity. ( 2.3 , 5.3 ) Hypertension: Monitor blood pressure during and after infusion as recommended. Withhold, reduce infusion rate, or discontinue based on severity. ( 2.3 , 5.4 ) Orthostatic Hypotension: Severe orthostatic hypotension, including cases requiring hospitalization, have occurred. Withhold, reduce dose, or discontinue based on severity. ( 2.3 , 5.5 ) Embryo-Fetal Toxicity: May cause fetal harm. Advise females of reproductive potential of potential risk to a fetus and to use effective contraception. ( 5.6 ) 5.1 Serious Infusion-Related Reactions DANYELZA can cause serious infusion reactions requiring urgent intervention including fluid resuscitation, administration of bronchodilators and corticosteroids, intensive care unit admission, infusion rate reduction or interruption of DANYELZA infusion. Infusion-related reactions included hypotension, bronchospasm, hypoxia, and stridor [see Adverse Reactions (6.1) ]. Serious infusion-related reactions occurred in 4% of patients in Study 201 and in 18% of patients in Study 12-230. Infusion-related reactions of any Grade occurred in 100% of patients in Study 201 and 94% of patients in Study 12-230. Hypotension of any grade occurred in 100% of patients in Study 201 and 89% of patients in Study 12-230.

The following clinically significant adverse reactions are also described elsewhere in the labeling: Serious Infusion-Related Reactions [see Warnings and Precautions (5.1) ] Neurotoxicity [see Warnings and Precautions (5.2) ] Myocarditis [see Warnings and Precautions (5.3) ] Hypertension [see Warnings and Precautions (5.4) ] Orthostatic Hypotension [see Warnings and Precautions (5.5) ] The most common adverse reactions (≥25%) are infusion-related reaction, pain, tachycardia, vomiting, cough, nausea, diarrhea, decreased appetite, hypertension, fatigue, erythema multiforme, peripheral neuropathy, urticaria, pyrexia, headache, injection site reaction, edema, anxiety, localized edema, and irritability ( 6.1 ). The most common Grade 3 or 4 laboratory abnormalities (≥5%) are decreased lymphocytes, decreased neutrophils, decreased hemoglobin, decreased platelet count, decreased potassium, increased alanine aminotransferase, decreased glucose, decreased calcium, decreased albumin, decreased sodium, and decreased phosphate ( 6.1 ). To report SUSPECTED ADVERSE REACTIONS, contact Y-mAbs Therapeutics, Inc, at 1-833-339-6227 (1-833-33YMABS), 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.

Lactation: Advise not to breastfeed. ( 8.2 ) 8.1 Pregnancy Risk Summary Based on its mechanism of action, DANYELZA may cause fetal harm when administered to pregnant women [see Clinical Pharmacology (12.1) ]. There are no available data on the use of DANYELZA in pregnant women and no animal reproduction studies have been conducted with DANYELZA. IgG1 monoclonal antibodies are transported across the placenta in a linear fashion as pregnancy progresses, with the largest amount transferred during the third trimester. Advise pregnant women of potential risk to a fetus. In the U.S. 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 There are no data on the presence of naxitamab-gqgk in human milk or its effects on the breastfed child, or on milk production, however, human IgG is present in human milk. Because of the potential for serious adverse reactions in a breastfed child from DANYELZA, advise women not to breastfeed during treatment and for 2 months after the last dose of DANYELZA. 8.3 Females and Males of Reproductive Potential DANYELZA may cause fetal harm when administered to a pregnant woman [see Use in Specific Populations (8.1) ].

Metabolism

The geometric mean (CV%) maximum plasma concentration (C max ) of naxitamab-gqgk was 57.4 µg/mL (49%) following DANYELZA 3 mg/kg intravenous infusion over 30 minutes. Elimination The mean terminal half-life of naxitamab-gqgk was 8.2 days.

• Nov 25, 2020BLABLA761171Y-mabs Therapeutics Inc

1. 1Hypotension6327%
2. 2Pain6126%
3. 3Hypertension3214%
4. 4Urticaria2912%
5. 5Off Label Use2812%
6. 6Rash2511%
7. 7Pyrexia229.4%
8. 8Oxygen Saturation Decreased177.3%
9. 9Bronchospasm156.4%
10. 10Hypoxia156.4%
11. 11Tachycardia156.4%
12. 12Abdominal Pain135.6%
13. 13Vomiting135.6%
14. 14Anaphylactic Reaction114.7%
15. 15Cough114.7%

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

• Study of Naxitamab and Sacituzumab Govitecan in Patients With Metastatic Triple-negative Breast Cancer (TNBC)
  Recruiting · Phase 1 · Phase 2 · Interventional · 31 enrolled · M.D. Anderson Cancer Center
  NCT07011654updated 2026-05-20
• Tipifarnib and Naxitamab for Relapsed/Refractory Neuroblastoma
  Recruiting · Phase 2 · Interventional · 98 enrolled · Giselle Sholler
  NCT06540963updated 2026-04-28
• Patients Between the Ages of 12 Months to 21 Years With Newly-Diagnosed High-Risk Neuroblastoma Will Receive Children's Oncology Group (COG) Type Recommended Therapy With the Addition of Naxitamab and Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) to Induction Cycles 1-5
  Not yet recruiting · Phase 2 · Interventional · 10 enrolled · Shaare Zedek Medical Center
  NCT07537400updated 2026-04-17
• Naxitamab for High-Risk Neuroblastoma Patients With Primary Refractory Disease or Incomplete Response to Salvage Treatment in Bone and/or Bone Marrow
  Recruiting · Phase 2 · Interventional · 122 enrolled · Y-mAbs Therapeutics
  NCT03363373updated 2026-02-19
• N10: A Study of Reduced Chemotherapy and Monoclonal Antibody (mAb)-Based Therapy in Children With Neuroblastoma
  Recruiting · Phase 2 · Interventional · 45 enrolled · Memorial Sloan Kettering Cancer Center
  NCT06528496updated 2026-02-10
• Naxitamab Added to Induction for Newly Diagnosed High-Risk Neuroblastoma
  Recruiting · Phase 2 · Interventional · 93 enrolled · Giselle Sholler
  NCT05489887updated 2026-02-03
• Combination Therapy of Antibody Hu3F8 With Granulocyte- Macrophage Colony Stimulating Factor (GM-CSF) in Patients With Relapsed/Refractory High-Risk Neuroblastoma
  Active not recruiting · Phase 1 · Phase 2 · Interventional · 186 enrolled · Memorial Sloan Kettering Cancer Center
  NCT01757626updated 2026-01-07
• Humanized Monoclonal Antibody 3F8 (Hu3F8) With Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) in the Treatment of Recurrent Osteosarcoma
  Active not recruiting · Phase 2 · Interventional · 46 enrolled · Memorial Sloan Kettering Cancer Center
  NCT02502786updated 2025-10-07
• A Study of the Effect of Hu3F8/GM-CSF Immunotherapy Plus Isotretinoin in Patients in First Remission of High-Risk Neuroblastoma
  Completed · Phase 2 · Interventional · 59 enrolled · Memorial Sloan Kettering Cancer Center
  NCT03033303updated 2025-10-07
• Quadruple Immunotherapy for Neuroblastoma
  Recruiting · Phase 2 · Interventional · 29 enrolled · Hong Kong Children's Hospital
  NCT05754684updated 2025-09-15

Frequently asked questions

How does Naxitamab work?

Naxitamab-gqgk binds to the glycolipid GD2. GD2 is a disialoganglioside that is overexpressed on neuroblastoma cells and other cells of neuroectodermal origin, including the central nervous system and peripheral nerves.

What is Naxitamab used for?

According to FDA labeling, Naxitamab carries indications including: DANYELZA is indicated, in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF), for the treatment of pediatric patients 1 year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy. This indication is approved under accelerated approval based on overall response rate and duration of response [see Clinical Studies (14) ] .. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Naxitamab?

Naxitamab is classified as Other monoclonal antibodies and antibody drug conjugates, Glycolipid Disialoganglioside-directed Antibody, Antibody-Surface Glycolipid Interactions, Glycolipid Disialoganglioside-directed Antibody Interactions.

What are the brand names for Naxitamab?

Naxitamab is marketed under brand names including Danyelza.

What are the contraindications for Naxitamab?

Naxitamab labeling lists contraindications including: DANYELZA is contraindicated in patients with a history of severe hypersensitivity reaction to naxitamab-gqgk. Reactions have included anaphylaxis [see Warnings and Precautions (5.1) ].. Always consult the full prescribing information and a clinician.