Drug updated on 12/11/2024
| Dosage Form | Injection (intravenous; 40 mg/10 mL [4 mg/mL]) |
| Drug Class | GD2-binding monoclonal antibodies |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- 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
Latest News
Summary
- This summary is based on the review of two randomized controlled trial(s). [1-2]
- In patients with long-chain fatty acid oxidation disorders (LC-FAODs), triheptanoin reduced major clinical events (MCEs) by 86% in the triheptanoin-naive cohort, decreasing the median rate from 2.00 to 0.28 events/patient/year (p = 0.0343). In the rollover cohort, there was a 43% reduction in MCEs, from 1.76 to 1.00 events/patient/year (p = 0.0347).
- In a long-term extension study, triheptanoin reduced the mean annualized MCE rate from 1.76 to 0.96 events/year (p = 0.0319) in the rollover group, with a 66% reduction in median MCE duration. The triheptanoin-naive group experienced an 80% reduction in MCE duration.
- In phosphofructokinase deficiency (PFKD), triheptanoin did not significantly improve exercise performance or heart rate, despite an increase in palmitate production and utilization during exercise.
- In the NCT02214160 (CL202) study, 68.1% of patients experienced treatment-emergent adverse events (TEAEs), mostly mild to moderate. Serious TEAEs, including diverticulitis, ileus, and rhabdomyolysis, were reported in five patients, but all resolved.
- In the long-term extension study, common adverse events included diarrhea, abdominal pain, and vomiting. Two patients had adverse events leading to death, but neither was drug-related.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Danyelza (naxitamab) Prescribing Information. | 2024 | Y-mAbs Therapeutics Inc., New York, NY |
Randomized Controlled Trials
| Document Title | Sex Distribution | Year | Source |
|---|---|---|---|
| Outpatient administration of naxitamab in combination with granulocyte-macrophage colony-stimulating factor in patients with refractory and/or relapsed high-risk neuroblastoma: Management of adverse events | Data not availableSubjects F: null% M: null% | 2023 | Cancer Reports (Hoboken, N.J.) |
| Survival Impact of Anti-GD2 Antibody Response in a Phase II Ganglioside Vaccine Trial Among Patients With High-Risk Neuroblastoma With Prior Disease Progression | 102Subjects F: 39% M: 61% | 2021 | Journal of Clinical Oncology |
Sex Distribution:
Year:
2023
Source:Cancer Reports (Hoboken, N.J.)
Sex Distribution:
F:39%
M:61%
102Subjects
Year:
2021
Source:Journal of Clinical Oncology