As a complement Factor B inhibitor, FABHALTA controls:

• Intravascular hemolysis (IVH)

• Extravascular hemolysis (EVH)

FABHALTA was studied to see how it could impact some of the most common challenges of paroxysmal nocturnal hemoglobinuria (PNH):

Hemoglobin Levels—Percent of people who:

• Had an increase of hemoglobin levels ≥ 2 g/dL

• Reached a hemoglobin level of ≥12 g/dL

Without the need for a red blood cell (RBC) transfusion

RBC transfusions—Percent of people who did not receive a red blood cell (RBC) transfusion during the clinical trials

The studies also looked at:

• Changes in hemoglobin levels over time 

• The rate of occurrences of major adverse vascular events involving the blood vessels such as stroke, heart attack, or blood clots

• The rate of clinical breakthrough hemolysis (in which a person experiences a decrease of ≥2 g/dL in hemoglobin compared to the last assessment or within 15 days, or other significant increase in PNH-related signs or symptoms) 

C5-Inhibitor Treatment–Experienced Adults With PNH

What was studied?

APPLY was a 24-week study of adults with PNH. It was meant to study the impact on people who switched to FABHALTA or continued on their C5 inhibitor (SOLIRIS® [eculizumab] or ULTOMIRIS® [ravulizumab-cwvz]).

Who was studied?

All 97 participants in APPLY were already on a C5 inhibitor for at least 6 months before the study and had hemoglobin levels below 10 g/dL as one of the key study criteria.

How was the study done?

Results were compared between 62 adults taking 200 mg of FABHALTA twice daily and 35 adults who remained on their C5 inhibitor (23 on SOLIRIS^® and 12 on  ULTOMIRIS^®).

Complement Inhibitor–Naive Adults With PNH

What was studied?

APPOINT was a 24-week study of FABHALTA in adults with PNH. It was meant to see the impact of FABHALTA on people who had never taken a complement inhibitor treatment before.

Who was studied?

All 40 participants in APPOINT had hemoglobin levels below 10 g/dL as one of the key study criteria.

How was the study done?

Results measured the impact of taking 200 mg of FABHALTA twice daily in 40 adults. In this trial, FABHALTA was not compared to another treatment. 

Hemoglobin increased by 2 or more g/dL in absence of red blood cell transfusions

• Data were assessed after 24 weeks

• After 24 weeks, FABHALTA increased hemoglobin levels by an average of 3.6 g/dL vs the average decrease of 0.1 g/dL with C5 inhibitors (SOLIRIS or ULTOMIRIS). The average starting hemoglobin level prior to treatment was 8.9 g/dL for both groups

The majority of people taking FABHALTA achieved normalized hemoglobin levels of ≥12 g/dL without the need for RBC transfusions after 24 weeks

• The average starting hemoglobin level prior to treatment start was 8.9 g/dL for both groups

Almost all who switched to FABHALTA did not receive an RBC transfusion

In the 6 months before the trial: 

• 57% (n=35/62) of people in the FABHALTA group had at least one RBC transfusion

• 60% (n=21/35) of people in the C5i group had at least one RBC transfusion 

Hemoglobin increased by 2 or more g/dL in the absence of red blood cell transfusions

• The average starting hemoglobin level prior to treatment was 8.2 g/dL

• Hemoglobin increases were assessed between Week 18 and Week 24

• Data based on Hb values from central laboratory

Hemoglobin levels

Percent of people on FABHALTA who reached a hemoglobin level of ≥12 g/dL in the absence of RBC transfusions

The data from this additional analysis are exploratory and presented for observation only. It is unknown if the following results were due to FABHALTA or not. We cannot make conclusions from exploratory data, but it is useful to help guide future research. 

• The average starting hemoglobin level prior to treatment was 8.2 g/dL

• Hemoglobin levels ≥ 12g/dL were assessed between Week 18 and Week 24

Red blood cell transfusions with FABHALTA

Percent of people on FABHALTA who did not receive a red blood cell transfusion between Weeks 2 and 24 of the clinical trial

The data from this additional analysis are exploratory and presented for observation only. It is unknown if the following results were due to FABHALTA or not. We cannot make conclusions from exploratory data, but it is useful to help guide future research. 

• 28 of 40 people (70%) required an RBC transfusion in the 6 months before starting the trial

ULTOMIRIS (ravulizumab-cwvz) and SOLIRIS (eculizumab) are registered trademarks of Alexion Pharmaceuticals, Inc.