pivatal-trial

Gamifant® (emapalumab-lzsg) was evaluated in a multicenter study1

The safety and efficacy of Gamifant was evaluated in a multicenter, open-label, single-arm study.

Safety

evaluated in 34 patients

safety-img

Efficacy

evaluated in 27 patients

  • were treatment-naïve prior to receiving Gamifant
  • received Gamifant after conventional treatments attempted

Study population1

Patients had refractory, recurrent, or progressive disease or were intolerant of 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%).

median-age

The median age of patients in the study was 1 year.

Patients ranged in age from 0.1 year to 13 years old.

vials-icon

Patients had received a median of 3 prior agents before enrollment.

Prior regimens included combinations of dexamethasone, etoposide, cyclosporine A, and antithymocyte globulin.

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 (i.e., 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