Increased Hb levels may help improve PNH symptoms due to anemia

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)

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 on SOLIRIS^® or ULTOMIRIS^® (35 of 62) had at least one RBC transfusion before switching to FABHALTA

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

• 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 the FACIT-Fatigue scale, a 13-item questionnaire used to measure fatigue and its impact on 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 1010 adults in the United States in 2002 and 2426 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 over-estimated 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 this 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)

These data are 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 a need for red blood cell transfusions

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

*Percentage is based on the number of people with Hb results at that time point.

These data are 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 after 24 weeks

In the 6 months before starting this study, 4% of people (2 of 52) needed RBC transfusions.

Before the study began and at various points during the study, people rated their fatigue using the FACIT-Fatigue scale, a 13-item questionnaire used to measure fatigue and its impact on 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 1010 adults in the United States in 2002 and 2426 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 study knew they were on FABHALTA, they may have under- or over-estimated their fatigue

• Before starting FABHALTA, about 70% of people rated their fatigue as "a little bit" or "not at all" for 11 of the 13 items in the questionnaire

• Because of the small number of people in the study (52) 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 Week 24 compared to the start of the study