FABHALTA was studied in both C5i-experienced (eculizumab or 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 of ≥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)
     - Lactate dehydrogenase (LDH) level

• Occurrence of major adverse vascular events (MAVEs)^¶

• Occurrence of clinical breakthrough hemolysis (BTH)^¶,#

• Safety^¶

^†Assessed between Day 126 and 168.^1
‡Assessed between Day 14 and 168. Requiring RBCs refers to any patient receiving transfusions or meeting protocol-defined criteria.^2
§Transfusion avoidance is defined as absence of administration of packed-RBC transfusions between Day 14 and Day 168.^1,4
IIExcludes values within 30 days post-transfusion in the randomized period.^1
¶Throughout the study.^2
#As per the protocol definition.²

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 data⁴
• 95% CIs were based on the Sato variance estimator⁴

PHASE 3, SINGLE-ARM, OPEN-LABEL, UNCONTROLLED STUDY^1,3,5

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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.^5
##Confirmed by at least 2 measurements 2 to 8 weeks apart during the screening period.⁵

PRIMARY END POINT¹

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

ADDITIONAL END POINTS⁵

• 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
     - ARC (10⁹/L)
     - LDH level

• Occurrence of MAVEs^¶

• Occurrence of BTH^¶,#

• Safety^¶

^†Assessed between Day 126 and 168.^1
‡Assessed between Day 14 and 168. Requiring RBCs refers to any patient receiving transfusions or meeting protocol-defined criteria.^5
§Transfusion avoidance is defined as absence of administration of packed-RBC transfusions between Day 14 and 168.^1,6
‖Excludes values within 30 days post-transfusion in the randomized period. ^5
¶Throughout the study.^5
#As per the protocol definition.⁵ 

Select baseline characteristics^1,5:

In the APPOINT study analysis:

• All additional end points were exploratory^1,6
• Unless otherwise noted, all end points were based on central lab data^1,6
• 95% CIs were based on the Clopper-Pearson method⁶