Emapalumab

Emapalumab-lzsg is a monoclonal antibody that binds to and neutralizes interferon gamma (IFNγ). Nonclinical data suggest that IFNγ plays a pivotal role in the pathogenesis of HLH by being hypersecreted.

• GAMIFANT is indicated for the treatment of: adult and pediatric patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. adult and pediatric patients with HLH/macrophage activation syndrome (MAS) in known or suspected Still’s disease, including systemic Juvenile Idiopathic Arthritis (sJIA), with an inadequate response or intolerance to glucocorticoids, or with recurrent MAS.

• None. None.contraindicated

For intravenous infusion only: Primary HLH recommended starting dosage: 1 mg/kg as an intravenous infusion over 1 hour twice per week. ( 2.1 ) HLH/MAS in Still’s disease recommended dosage schedule: 6 mg/kg, followed by 3 mg/kg every 3 days for 5 doses, then 3 mg/kg twice per week. Given as an intravenous infusion over 1 hour. ( 2.2 ) For both indications, the dose may be titrated up to a maximum of 10 mg/kg. ( 2.5 , 2.6 ) 2.1 Recommended Dosage for Primary Hemophagocytic Lymphohistiocytosis The recommended starting dose of GAMIFANT is 1 mg/kg given as a central or peripheral intravenous infusion over 1 hour twice per week (every three to four days). Doses subsequent to the initial dose may be increased based on clinical and laboratory criteria [see Dosage and Administration ( 2.5 )] . Administer GAMIFANT until hematopoietic stem cell transplantation (HSCT) is performed or unacceptable toxicity. Discontinue GAMIFANT when a patient no longer requires therapy for the treatment of HLH. 2.2 Recommended Dosage for Hemophagocytic Lymphohistiocytosis/Macrophage Activation Syndrome GAMIFANT is administered as a central or peripheral intravenous infusion over 1 hour according to the dosage schedule in Table 1. Doses may be increased based on clinical and laboratory criteria [see Dosage and Administration ( 2.6 )] . Discontinue GAMIFANT when a patient no longer requires therapy for the treatment of HLH/MAS. Table 1.

Infections: Monitor patients for signs and symptoms and treat promptly. Test for latent tuberculosis. Consider administering prophylactic treatment against herpes zoster, Pneumocystis jirovecii and fungal infections. ( 5.1 ) Live Vaccines: Do not administer live or live attenuated vaccines to patients receiving GAMIFANT. ( 5.2 ) Infusion-Related Reactions: Monitor patients for infusion-related reactions. Interrupt infusion for infusion reactions and institute appropriate medical management. ( 5.3 ) 5.1 Infections GAMIFANT may increase the risk of fatal and serious infections to include specific pathogens favored by IFNγ neutralization, including mycobacteria, herpes zoster virus, and Histoplasma capsulatum . Do not administer GAMIFANT in patients with infections caused by these pathogens until appropriate treatment has been initiated. In patients with primary HLH receiving GAMIFANT in clinical trials, serious infections such as sepsis, pneumonia, bacteremia, disseminated histoplasmosis, necrotizing fasciitis, viral infections, and perforated appendicitis were observed in 32% of patients. The reported infections were viral (41%), bacterial (35%), fungal (9%), and the pathogen was not identified in 15% of cases. In patients with HLH/MAS in Still’s disease receiving GAMIFANT in clinical trials, serious infections such as pneumonia, cytomegalovirus infection, cytomegalovirus infection reactivation, and sepsis were observed in 13% of patients. The reported infections were viral (44%), bacterial (13%), fungal (3%) and the pathogen was not identified in (13%) of patients.

The following adverse reactions are described elsewhere in the labeling: Infections [see Warnings and Precautions ( 5.1 )] Increased Risk of Infection with Use of Live Vaccines [see Warnings and Precautions ( 5.2 )] Infusion-Related Reactions [see Warnings and Precautions ( 5.3 )] In patients with primary HLH, the most common adverse reactions (≥ 20%) were: infections, hypertension, infusion-related reactions, and pyrexia. ( 6.1 ) In patients with HLH/MAS in Still’s disease, the most common adverse reactions (≥20%) were: viral infections, including cytomegalovirus infection or reactivation, and rash. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact 1-866-773-5274 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trial 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. Primary HLH The safety data described in this section reflect exposure to GAMIFANT in which 34 patients with untreated primary HLH and previously treated patients with primary HLH ( NCT01818492 ) received GAMIFANT at a starting dose of 1 mg/kg every 3 days with dose increases up to 10 mg/kg [see Dosage and Administration ( 2.1 ) and Clinical Studies ( 14 )] . The median duration of treatment with GAMIFANT was 59 days (range: 4 to 245 days) and the median cumulative dose was 25 mg/kg (range: 4 to 254 mg/kg).

8.1 Pregnancy Risk Summary There are no available data on GAMIFANT use in pregnant women to inform a drug-associated risk of adverse developmental outcomes. In an animal reproduction study, a murine surrogate anti-mouse IFNγ antibody administered to pregnant mice throughout gestation crossed the placental barrier, and no fetal harm was observed (see Data ) . The estimated background risk of major birth defects and miscarriage for the indicated population is unknown. All pregnancies have a background risk of birth defect, loss, or other adverse outcomes. 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. Data Animal Data In a mouse embryo-fetal development study, a murine surrogate anti-mouse IFNγ antibody was administered every 3-4 days throughout organogenesis and late gestation at doses of 0, 30, 75 or 150 mg/kg/occasion. The surrogate antibody was detected in the plasma of all treated pregnant mice and their corresponding fetuses. No maternal toxicity occurred and there was no evidence of teratogenicity or effects on embryo-fetal survival or growth. 8.2 Lactation Risk Summary There is no information regarding the presence of emapalumab-lzsg in human milk, the effects on the breastfed child, or the effects on milk production.

Metabolism

The pharmacokinetics of emapalumab-lzsg were evaluated in healthy adult subjects and in patients with HLH. Following a 1 mg/kg emapalumab-lzsg dose, median steady state peak concentration was 44 mcg/mL, which was 2.9 times higher than after the first dose.

Consider contacting the Poison Help line (1-800-222-1222) or a medical toxicologist for additional overdose management recommendations.

• Nov 20, 2018BLABLA761107Novimmune S.a.

1. 1Off Label Use8043%
2. 2Haemophagocytic Lymphohistiocytosis4826%
3. 3Condition Aggravated3418%
4. 4Death2212%
5. 5Septic Shock1910%
6. 6Drug Ineffective168.7%
7. 7Epstein-barr Virus Infection147.6%
8. 8Adenovirus Infection137.1%
9. 9Pyrexia94.9%
10. 10Respiratory Failure94.9%
11. 11Nervous System Disorder84.3%
12. 12Colitis73.8%
13. 13Disseminated Intravascular Coagulation73.8%
14. 14Gastrointestinal Haemorrhage73.8%
15. 15Serum Ferritin Increased73.8%

The 10 most recently updated of 29 ClinicalTrials.gov registrations naming Emapalumab as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• A Study of Treatment of Inflammation Before Stem Cell Transplant in People With a Primary Immune Regulatory Disorder (PIRD) and/or an Autoinflammatory Condition
  Recruiting · Phase 2 · Interventional · 39 enrolled · Joseph Oved
  NCT05787574updated 2026-06-02
• A Pilot Study of Emapalumab for the Treatment of CAR T-Cell Therapy-Associated Prolonged Cytopenia
  Recruiting · Phase 2 · Interventional · 32 enrolled · M.D. Anderson Cancer Center
  NCT06285825updated 2026-05-18
• Allogeneic Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease (CGD) With an Alemtuzumab, Busulfan and TBI-based Conditioning Regimen Combined With Cytokine (IL-6, +/- IFN-gamma) Antagonists
  Recruiting · Phase 1 · Phase 2 · Interventional · 50 enrolled · National Institute of Allergy and Infectious Diseases (NIAID)
  NCT05463133updated 2026-05-14
• Emapalumab With Post-Transplant Cyclophosphamide, Tacrolimus and Mycophenolate Mofetil for the Prevention of Graft-versus-Host Disease After Donor Reduced-Intensity Hematopoietic Cell Transplant
  Recruiting · Phase 1 · Interventional · 15 enrolled · City of Hope Medical Center
  NCT06996119updated 2026-05-11
• Emapalumab Prophylaxis of Bispecific T-Cell Engagers (BiTEs) Associated CRS and ICANS
  Not yet recruiting · Phase 2 · Interventional · 60 enrolled · NYU Langone Health
  NCT07567014updated 2026-05-06
• Targeting Interferon Gamma With Emapalumab to Lung Transplant Recipients With Interferon Gamma-high Acute Lung Allograft Dysfunction
  Not yet recruiting · Phase 2 · Interventional · 40 enrolled · University of California, San Francisco
  NCT07538336updated 2026-04-20
• Emapalumab MDA5 Rapidly Progressive Interstitial Lung Disease (RP-ILD) Study
  Not yet recruiting · Phase 2 · Interventional · 5 enrolled · University of Miami
  NCT07486869updated 2026-04-09
• A Post-authorization Study to Describe the Safety and Efficacy of Emapalumab for the Treatment of pHLH in Treatment Experienced Chinese Patients
  Completed · Phase 4 · Interventional · 13 enrolled · Swedish Orphan Biovitrum
  NCT05744063updated 2026-03-13
• First-in-human Study of Interferon-y PET Imaging to Assess Response to Immunotherapy
  Recruiting · Phase 1 · Interventional · 6 enrolled · Nerissa T. Viola
  NCT06439914updated 2026-03-11
• Evaluate Efficacy, Safety and Tolerability, PK and PD of Emapalumab in Children and Adults With MAS in Still's or SLE
  Completed · Phase 3 · Interventional · 33 enrolled · Swedish Orphan Biovitrum
  NCT05001737updated 2026-02-19

Frequently asked questions

How does Emapalumab work?

Emapalumab-lzsg is a monoclonal antibody that binds to and neutralizes interferon gamma (IFNγ). Nonclinical data suggest that IFNγ plays a pivotal role in the pathogenesis of HLH by being hypersecreted.

What is Emapalumab used for?

According to FDA labeling, Emapalumab carries indications including: GAMIFANT is indicated for the treatment of: adult and pediatric patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. adult and pediatric patients with HLH/macrophage activation syndrome (MAS) in known or suspected Still’s disease, including systemic Juvenile Idiopathic Arthritis (sJIA), with an inadequate response or intolerance to glucocorticoids, or with recurrent MAS.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Emapalumab?

Emapalumab is classified as Monoclonal antibodies, Interferon gamma Blocker, Antibody-Receptor Interactions, Interferon gamma Antagonists.

What are the brand names for Emapalumab?

Emapalumab is marketed under brand names including Gamifant.

What are the contraindications for Emapalumab?

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