APPLY: FABHALTA was studied in C5i-experienced adults with PNH
PHASE 3, OPEN-LABEL, ACTIVE COMPARATOR-CONTROLLED STUDY1-3
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*Randomization was stratified based on prior C5i treatment and transfusion history within the last 6 months.1
PRIMARY END POINTS1
Proportion of patients achieving sustained Hb increase ≥2 g/dL† from baseline without a need for RBC transfusions‡ after 24 weeks
Proportion of patients achieving sustained Hb level of ≥12 g/dL† without a need for RBC transfusions after 24 weeks‡
ADDITIONAL END POINTS1,2
RBC transfusion avoidance§
Change from baseline† in:
• Hb levels (g/dL)‖
• FACIT-Fatigue scores
• Absolute reticulocyte count (ARC) (109/L)
• LDH levelOccurrence of major adverse vascular events¶
Occurrence of clinical breakthrough hemolysis¶,#
†Assessed between Days 126 and 168.1
‡Assessed between Days 14 and 168. Requiring RBCs refers to any patient receiving transfusions or meeting protocol-defined criteria.2,4
§Transfusion avoidance is defined as absence of administration of packed-RBC transfusions between Days 14 and 168.1,4
‖Excludes values within 30 days post-transfusion.1
¶Throughout the study.2,4
#As per the protocol definition.2,4
Select baseline characteristics1,2:
Baseline disease characteristics were generally well balanced between treatment groups.