Indication
Gamifant® (emapalumab-lzsg) is an interferon gamma (IFNγ)–blocking antibody 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.
Important Safety Information
Infections
Before initiating Gamifant, patients should be evaluated for infection, including latent tuberculosis (TB)... Prophylaxis for TB should be administered to patients who are at risk for TB or known to have a positive purified protein derivative (PPD) test result or positive IFNγ release assay.
Indication
Gamifant® (emapalumab-lzsg) is an interferon gamma (IFNγ)–blocking antibody 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.
Important Safety Information
Infections
Before initiating Gamifant, patients should be evaluated for infection, including latent tuberculosis (TB). Prophylaxis for TB should be administered to patients who are at risk for TB or known to have a positive purified protein derivative (PPD) test result or positive IFNγ release assay.
During Gamifant treatment, patients should be monitored for TB, adenovirus, Epstein-Barr virus (EBV), and cytomegalovirus (CMV) every 2 weeks and as clinically indicated.
Patients should be administered prophylaxis for herpes zoster, Pneumocystis jirovecii, and fungal infections prior to Gamifant administration.
Increased Risk of Infection With Use of Live Vaccines
Do not administer live or live attenuated vaccines to patients receiving Gamifant and for at least 4 weeks after the last dose of Gamifant. The safety of immunization with live vaccines during or following Gamifant therapy has not been studied.
Infusion-Related Reactions
Infusion-related reactions, including drug eruption, pyrexia, rash, erythema, and hyperhidrosis, were reported with Gamifant treatment in 27% of patients. In one-third of these patients, the infusion-related reaction occurred during the first infusion.
Adverse Reactions
In the pivotal trial, the most commonly reported adverse reactions (≥10%) for Gamifant included infection (56%), hypertension (41%), infusion-related reactions (27%), pyrexia (24%), hypokalemia (15%), constipation (15%), rash (12%), abdominal pain (12%), CMV infection (12%), diarrhea (12%), lymphocytosis (12%), cough (12%), irritability (12%), tachycardia (12%), and tachypnea (12%).
Additional selected adverse reactions (all grades) that were reported in less than 10% of patients treated with Gamifant included vomiting, acute kidney injury, asthenia, bradycardia, dyspnea, gastrointestinal hemorrhage, epistaxis, and peripheral edema.
Click here for full Prescribing Information for Gamifant.
You may also contact Sobi at medinfo.us@sobi.com or 866-773-5274.
References
- Jordan MB, Allen CE, Weitzman S, Filipovich AH, McClain KL. How I treat hemophagocytic lymphohistiocytosis. Blood. 2011;118(15):4041-4052. doi:10.1182/blood-2011-03-278127
- Zhang K, Filipovich AH, Johnson J, et al. Hemophagocytic lymphohistiocytosis, familial. March 22, 2006. Updated September 30, 2021. Accessed March 29, 2024. https://www.ncbi.nlm.nih.gov/books/NBK1444/?report=classic
- Locatelli F, Jordan MB, Allen C, et al. Emapalumab in children with primary hemophagocytic lymphohistiocytosis. N Engl J Med. 2020;382(19):1811-1822. doi:10.1056/NEJMoa1911326
- Stone JH, McDowell PJ, Jayne DRW, et al. The glucocorticoid toxicity index: measuring change in glucocorticoid toxicity over time. Semin Arthritis Rheum. 2022;55:152010. doi:10.1016/j.semarthrit.2022.152010
- La Rosée P. Alleviating the storm: ruxolitinib in HLH. Blood. 2016;127(13):1626-1627. doi:10.1182/blood-2016-02-697151
- De Benedetti F, Prencipe G, Bracaglia C, Marasco E, Grom AA. Targeting interferon-γ in hyperinflammation: opportunities and challenges. Nat Rev Rheumatol. 2021;17(11):678-691. doi:10.1038/s41584-021-00694-z
- Gamifant (emapalumab-lszg) prescribing information. Stockholm, Sweden: Sobi, Inc. 2022.
- Morimoto A, Nakazawa Y, Ishii E. Hemophagocytic lymphohistiocytosis: pathogenesis, diagnosis, and management. Pediatr Int. 2016;58(9):817-825. doi:10.1111/ped.13064
- FDA approves first treatment specifically for patients with rare and life-threatening type of immune disease [news release]. Silver Spring, MD: Food and Drug Administration; November 20, 2018. Accessed March 29, 2024. https://www.fda.gov/newsevents/newsroom/pressannouncements/ucm626263.htm
- Sepulveda FE, de Saint Basile G. Hemophagocytic syndrome: primary forms and predisposing conditions. Curr Opin Immunol. 2017;49:20-26. doi:10.1016/j.coi.2017.08.004
Getting patients to transplant
For patients with primary hemophagocytic lymphohistiocytosis (HLH), a successful hematopoietic stem cell transplantation (HSCT) is the only cure. Before HSCT can be performed, prompt and effective treatment is necessary to calm hyperinflammatory symptoms.1,2
Primary HLH treatment has 3 main goals:
Stabilize the patient
Subdue hyperinflammation to prevent irreversible organ damage1
Minimize treatment toxicities
Reduce collateral damage of broad-spectrum medications3,4
Prepare for transplant
Condition the patient for HSCT, the only curative treatment for primary HLH1
The treatment landscape
Broadly acting conventional treatment options do not target interferon gamma (IFNγ), a key driver of hyperinflammation in primary HLH. Instead, these therapies seek to control hyperinflammation through broad immune suppression.5,6
There has been a clear need for a targeted option with an established safety profile to treat patients with primary HLH.1
A targeted Treatment
Gamifant is the first and only treatment designed specifically for primary HLH. It works by targeting IFNγ, a central and upstream cytokine in the pathogenesis of the disease.3,7-9
See how it worksReal patient cases
See how targeted therapy with Gamifant helped 2 patients with primary HLH prepare for HSCT.
