thogens favored by IFNγneutralization at the time of GAMIFANT initiation.
The efficacy of GAMIFANT was based upon overall response rate (ORR) at the end oftreatment, defined as achievement of either a complete or partial response or HLH improvement.
ORR was eva luated using an algorithm that included the following objective clinical andlaboratory parameters: fever, splenomegaly, central nervous system symptoms, complete bloodcount, fibrinogen and/or D-dimer, ferritin, and soluble CD25 (also referred to as solubleinterleukin-2 receptor) levels. Complete response was defined as normalization of all HLHabnormalities (i.e., no fever, no splenomegaly, neutrophils > 1x109/L, platelets > 100x109/L,ferritin < 2,000 g/L, fibrinogen > 1.50 g/L, D-dimer < 500 g/L, normal CNS symptoms, noworsening of sCD25 > 2-fold baseline). Partial response was defined as normalization of ≥ 3
HLH abnormalities. HLH improvement was defined as ≥ 3 HLH abnormalities improved by atleast 50% from baseline.
Table 4: Overall Response Rate at End of Treatment
GAMIFANT
(N=27)
Overall Response Rate
N (%) 17 (63)
(95% CI) (0.42, 0.81)
p-value† 0.013
Overall Response by Category
Complete response, n (%) 7 (26)
Partial response 8 (30)
HLH improvement 2 (7.4)
†p-value based on Exact Binomial Test at a one-sided significance level of 2.5% comparing proportion of patientswith overall response to hypothesized null hypothesis of 40%.
CI = confidence interval
The median duration of first response, defined as time from achievement of first response to lossof first response, is not reached (range: 4-56+ days). Seventy percent (19/27) of patients
proceeded to HSCT.
16 HOW SUPPLIED/STORAGE AND HANDLING
GAMIFANT (emapalumab-lzsg) injection is a sterile, clear to slightly opalescent, colorless toslightly yellow solution supplied in the following packaging configuration:
NDC 72171-501-01 – containing one 10 mg/2 mL (5 mg/mL) single-dose vial
NDC 72171-505-01 – containing one 50 mg/10 mL (5 mg/mL) single-dose vial
Store GAMIFANT in a refrigerator at 2ºC to 8ºC (36ºF to 46ºF) in original carton to protect fromlight.
Do not freeze or shake. This product contains no preservative.
17 PATIENT COUNSELING INFORMATION
Advise the patient to read the FDA-approved patient labeling (Medication Guide).
Infections
Inform patients and their caregivers of the risk of developing infections during treatment withGAMIFANT, and to report any symptoms of infection [see Warnings and Precautions (5.1)].
Vaccinations
Advise patients and their caregivers that the patient should not receive live or live attenuatedvaccines during GAMIFANT treatment [see Warnings and Precautions (5.2)].
Infusion-Related Reactions
Advise patients and their caregivers of the potential for developing infusion-related reactionsduring treatment with GAMIFANT [see Warnings and Precautions (5.3)].
Manufactured by:
Novimmune SA
Geneva, Switzerland
U.S. License Number 2082
Distributed by:
Sobi Inc.
890 Winter Street
Waltham, MA 02451
Manufactured at:
Patheon Italia S.p.A
2° Trav. SX Via Morolense, 5
03013-Ferentino Italy
Product of the United Kingdom
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