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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.


evaluated in 34 patients



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.