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.
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References
- Gamifant [prescribing information]. Stockholm, Sweden: Swedish Orphan Biovitrum AB.
- Sepulveda F, de Saint Basile G. Hemophagocytic syndrome: primary forms and predisposing conditions. Curr Opin Immunol. 2017; 49:20-26. http://dx.doi.org/10.1016/j.coi.2017.08.004.
- Jordan MB, Allen CE, Weitzman S, Filipovich AH, McClain KL. How I treat hemophagocytic lymphohistiocytosis. Blood. 2011;118(15):4041-4052. doi: https://doi.org/10.1182/blood-2011-03-278127.
- Lehmberg K, Nichols KE, Henter J-I, et al. Consensus recommendations for the diagnosis and management of hemophagocytic lymphohistiocytosis associated with malignancies. Haematologica. 2015:100(8):997-1004.
- Marsh RA, Haddad E. How I treat primary haemophagocytic lymphohistiocytosis. Br J Haematol. 2018;182(2):185-199. doi: 10.1111/bjh.15274.
Gamifant® (emapalumab-lzsg) demonstrated efficacy and safety in a pivotal trial1
The safety and efficacy of Gamifant was evaluated in a multicenter, open-label, single-arm study.
Safety
evaluated in 34 patients
Efficacy
evaluated in 27 patients
- Were treatment-naïve prior to receiving Gamifant
- Received Gamifant after conventional treatments attempted
Study population1
Gamifant was studied in patients with significant unmet need (n=27), including patients with primary HLH who had refractory, recurrent, or progressive disease or intolerance to conventional therapy.
A genetic diagnosis of primary HLH was confirmed in 82% of patients.
The most frequent causative mutations were FHL3-UNC13D (MUNC 13-4) (26%), FHL2-PRF1 (19%), and Griscelli syndrome type 2 (19%).1
The median age of patients in the study was 1 year.
Patients ranged in age from 0.2 years to 13 years old.1
Patients had received a median of 3 prior agents before enrollment.
Prior regimens included combinations of dexamethasone, etoposide, cyclosporine A, and antithymocyte globulin.1
The median duration of Gamifant treatment was 59 days.
Duration of treatment ranged from 4 to 245 days in 34 patients studied for safety.1
Primary endpoint1
The primary endpoint was overall response rate (ORR) at end of treatment. ORR was defined as achievement of either complete or partial response or HLH improvement and evaluated using an algorithm of objective clinical and laboratory parameters.
Complete response
defined as normalization of all HLH abnormalities (ie, no fever, no splenomegaly, neutrophils >1x109/L, platelets >100x109/L, ferritin <2000 μg/L, fibrinogen >1.50 g/L, D-dimer <500 μg/L, normal CNS symptoms, no worsening of soluble CD25* >2-fold baseline)
*Soluble CD25 is also referred to as soluble interleukin-2 receptor.
Partial response
defined as normalization of
≥3 HLH abnormalities
HLH improvement
defined as ≥3 HLH abnormalities improved by at least 50% from baseline
see the results
*Soluble CD25 is also referred to as soluble interleukin-2 receptor.