Drug updated on 12/11/2024
| Dosage Form | Injection (intravenous; 10 mg/2 mL [5 mg/mL], 50 mg/10 mL [5 mg/mL], 100 mg/20 mL [5 mg/mL], 50 mg/2 mL [25 mg/mL], 100 mg/4 mL [25 mg/mL]) |
| Drug Class | Interferon gamma (IFNγ) blocking antibodies |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for the treatment of adult and pediatric (newborn and older) patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy.
Latest News
Summary
- This summary is based on the review of two randomized controlled trial(s). [1-2]
- Emapalumab significantly reduced interferon gamma (IFNgamma) activity, correlating with improvements in clinical and laboratory outcomes in patients with primary haemophagocytic lymphohistiocytosis (pHLH), using a dosing regimen starting at 1 mg/kg with potential increases based on disease activity.
- The overall response rate to emapalumab was 63% in previously treated patients and 65% in all patients, both surpassing the prespecified null hypothesis of 40% (P = 0.02 for previously treated patients and P = 0.005 for all patients). Additionally, 70% of previously treated patients and 65% of all patients were able to proceed to transplantation.
- Emapalumab was not associated with organ toxicity, demonstrating a favorable safety profile in terms of organ-specific adverse effects.
- Severe infections occurred in 10 patients during treatment, with one patient discontinuing due to disseminated histoplasmosis.
- The study population included patients aged 18 years or younger with primary haemophagocytic lymphohistiocytosis (pHLH), with no clinically significant differences in response rates between previously treated patients (63%) and all patients receiving emapalumab (65%), and comparable survival rates (74% vs. 71%) and transplantation eligibility (70% vs. 65%) between these groups.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Gamifant (emapalumab-lzsg) Prescribing Information. | 2024 | Sobi, Inc., Waltham, MA |
Randomized Controlled Trials
| Document Title | Sex Distribution | Year | Source |
|---|---|---|---|
| Emapalumab in primary haemophagocytic lymphohistiocytosis and the pathogenic role of interferon gamma: A pharmacometric model-based approach | 33Subjects F: 55% M: 45% | 2022 | British Journal of Clinical Pharmacology |
| Emapalumab in Children with Primary Hemophagocytic Lymphohistiocytosis | 34Subjects F: 53% M: 47% | 2020 | The New England Journal of Medicine |
Sex Distribution:
F:55%
M:45%
33Subjects
Year:
2022
Source:British Journal of Clinical Pharmacology
Sex Distribution:
F:53%
M:47%
34Subjects
Year:
2020
Source:The New England Journal of Medicine
Clinical Practice Guidelines
| Document Title | Year | Source |
|---|---|---|
| Recommendations for the management of hemophagocytic lymphohistiocytosis in adults | 2019 | Blood |
| Challenges in the diagnosis of hemophagocytic lymphohistiocytosis: Recommendations from the North American Consortium for Histiocytosis (NACHO) | 2019 | Pediatric Blood & Cancer |