97 adults with PNH who had been on a C5 inhibitor (SOLIRIS^® or ULTOMIRIS^®) for ≥6 months before the study and had hemoglobin (Hb) levels of <10 g/dL

In the 24-week study:

• 62 adults switched to FABHALTA

• 35 adults remained on SOLIRIS^® or ULTOMIRIS^® (23 on SOLIRIS^® and 12 on ULTOMIRIS^®)

The effect of switching to FABHALTA (200 mg, oral capsule twice daily) after receiving SOLIRIS^® or ULTOMIRIS^®

*Normal Hb levels vary, but are generally between 12-16 g/dL for women and 13-18 g/dL for men.
^†Breakthrough hemolysis was defined as a decrease of ≥2 g/dL in Hb when compared to the latest assessment, or any significant PNH-related signs, symptoms, and other lab criteria.

40 adults with PNH who had hemoglobin (Hb) levels <10 g/dL as one of the key study criteria.

The effect of FABHALTA (200 mg, oral capsule twice daily) on adults with PNH who had never received a complement inhibitor. FABHALTA was not compared to another treatment in this study.

*Normal Hb levels vary, but are generally between 12-16 g/dL for women and 13-18 g/dL for men.
^†Breakthrough hemolysis was defined as a decrease of ≥2 g/dL in Hb when compared to the latest assessment, or any significant PNH-related signs, symptoms, and other lab criteria.

Average change in Hb from the start of study

People who continued on FABHALTA maintained an increase in Hb levels, and those who switched to FABHALTA at Week 24 increased their Hb levels.

• 3.3 g/dL average increase in Hb levels at Week 48 compared to the start of the study^† for people who continued taking FABHALTA (57 of 62)

• 3.5 g/dL average increase in Hb levels at Week 48 compared to the start of the study^† for people who switched from SOLIRIS^® or ULTOMIRIS^® to FABHALTA (29 of 35)

*Average was assessed between Weeks 18 and 24. Hb levels within 30 days of a red blood cell (RBC) transfusion were excluded.
^†Average was assessed at Week 48. Hb levels within 30 days of an RBC transfusion were excluded.

Percentage of people who didn’t need RBC transfusions between Weeks 2 and 24

In the 6 months before starting this study:

• 57% of people who switched to FABHALTA from SOLIRIS^® or ULTOMIRIS^® (35 of 62) had at least one RBC transfusion

• 60% of people who continued on SOLIRIS^® or ULTOMIRIS^® (21 of 35) had at least one RBC transfusion

• 92% of people who continued on FABHALTA (57 of 62) didn’t need RBC transfusions between Weeks 2 and 48*

• 94% of people who switched at Week 24 from SOLIRIS^® or ULTOMIRIS^® to FABHALTA (32 of 34) didn’t need RBC transfusions between Weeks 26 and 48*

*Transfusion avoidance was defined as people who didn't receive RBC transfusions during the specified time period.

Before the study began and at various points during the study, people rated their fatigue using FACIT-Fatigue scale, a 13-item questionnaire used to measure fatigue and its impact on their daily activities and function.

FACIT-Fatigue scores range from 0 to 52

*The average FACIT (Functional Assessment of Chronic Illness Therapy)-Fatigue score for the general population was determined in a study of 1,010 adults in the United States in 2002 and 2,426 adults in Germany in 2018.

Fatigue was studied with FABHALTA and SOLIRIS^® or ULTOMIRIS^®

Data from this analysis describe how people reported their fatigue. There are important limitations to the data presented below that you should consider:

• Because people knew whether they were on FABHALTA or SOLIRIS^® or ULTOMIRIS^®, they may have under- or overestimated their fatigue

• While on SOLIRIS^® or ULTOMIRIS^® and before starting FABHALTA, about half the people rated their fatigue as “a little bit” and “not at all” for 10 of the 13 items in the questionnaire

• Because of the small number of people in the study (97), the low level of fatigue reported before taking FABHALTA, and since people knew which treatment they were on, it is unknown if these results were due to FABHALTA and no conclusions can be made about the effect of FABHALTA on fatigue

• No conclusions or comparisons between FABHALTA and SOLIRIS^® or ULTOMIRIS^® can be made based on these data

Average change in FACIT-Fatigue score at Week 24^† compared to the start of the study

Average change in FACIT-Fatigue score at Week 48 compared to the start of the study

• +9.8 points for people who continued on FABHALTA (55 of 62)

• +10.96 points for people who switched at Week 24 from SOLIRIS^® or ULTOMIRIS^® to FABHALTA (26 of 33)

^†FACIT-Fatigue score was assessed between Weeks 18 and 24. Values within 30 days after transfusion were included in the analysis.

This additional analysis is presented for observation only. It is unknown if the following results were due to FABHALTA. We cannot make conclusions from these data, but they are useful to help guide future research.

Percentage of people who maintained Hb levels of ≥12 g/dL^* between Weeks 18 and 24 without red blood cell (RBC) transfusions

• The average Hb level at the start of the study was 8.2 g/dL, which is below normal Hb levels

• Normal Hb levels vary, but are generally between 12-16 g/dL for women and 13-18 g/dL for men

78% of people on FABHALTA (31 of 40) maintained Hb levels of ≥12 g/dL* without the need for RBC transfusions between Weeks 2 and 48.

This additional analysis is presented for observation only. It is unknown if the following results were due to FABHALTA. We cannot make conclusions from these data, but they are useful to help guide future research.

Percentage of people who didn’t need RBC transfusions between Weeks 2 and 24

In the 6 months before starting this study, 70% of people (28 of 40) needed RBC transfusions.

98% of people on FABHALTA (39 of 40) did not need RBC transfusions between Weeks 2 and 48.

Before the study began and at various points during the study, people rated their fatigue using FACIT-Fatigue scale, a 13-item questionnaire used to measure fatigue and its impact on their daily activities and function.

FACIT-Fatigue scores range from 0 to 52

^*The average FACIT (Functional Assessment of Chronic Illness Therapy)-Fatigue score for the general population was determined in a study of 1,010 adults in the United States in 2002 and 2,426 adults in Germany in 2018.

Fatigue was studied with FABHALTA

Data from this analysis describe how people reported their fatigue. There are important limitations to the data presented below that you should consider:

• Because people in the trial knew they were on FABHALTA, they may have under- or over-estimated their fatigue

• Because of the small number of people in the trial (40) and the fact that people knew the medicine they were taking, it is unknown if these results were due to FABHALTA and no conclusions can be made about the effect of FABHALTA on fatigue

Average change in FACIT-Fatigue score at 24 weeks^† compared to the start of the study

Average change in FACIT-Fatigue score at Week 48 compared to the start of the study

• +10.9 points for people who continued taking FABHALTA (39 of 40)

^†FACIT-Fatigue score was assessed between Weeks 18 and 24.