FABHALTA was studied in both C5i-experienced (eculizumab and ravulizumab) and complement inhibitor–naive adults with PNH

PHASE 3, OPEN-LABEL, ACTIVE COMPARATOR-CONTROLLED STUDY^1-3

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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 POINTS^1,2

• RBC transfusion avoidance^§ 

• Change from baseline^† in:    
  •  Hb levels (g/dL)^‖   
  •  FACIT-Fatigue scores    
  •  Absolute reticulocyte count (ARC) (10⁹/L)    
  •  LDH level

• Occurrence 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 characteristics^1,2:

Baseline disease characteristics were generally well balanced between treatment groups.

In the APPLY study analysis:

• All end points were based on central laboratory data^1,5
• 95% CIs were based on the Sato variance estimator⁵

PHASE 3, SINGLE-ARM, OPEN-LABEL, UNCONTROLLED STUDY^1,3,4

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**Confirmed by 2 measurements 2 to 8 weeks apart for patients not receiving an RBC transfusion during screening, or by 1 measurement during the first screening visit for patients receiving an RBC transfusion.^{4     
††}Confirmed by 2 measurements 2 to 8 weeks apart during the screening period.⁴

PRIMARY END POINT^1,4

• Proportion of patients achieving sustained Hb increase  ≥2 g/dL^† from baseline without a need for RBC transfusions^‡ after 24 weeks

ADDITIONAL END POINTS⁴

• Proportion of patients with sustained Hb level of ≥12 g/dL^† without a need for RBC transfusions^‡

• RBC transfusion avoidance^§

• Change from baseline^† in:       
  •  Hb levels (g/dL)^‖       
  •  FACIT-Fatigue scores       
  •  Absolute reticulocyte count (ARC) (10⁹/L)       
  •  LDH level

• Occurrence 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  
‖Excludes values within 30 days post-transfusion.^1  
¶Throughout the study.^2,4  
#As per the protocol definition.^2,4 

Select baseline characteristics^1,4:

In the APPOINT study analysis:

• All additional end points were exploratory^1,5
• Unless otherwise noted, all end points were based on central lab data^1,5
• 95% CIs were based on the Clopper-Pearson method⁵