If you're like Angelo, an adult with PNH taking EMPAVELI,
Where will you go next?
Talk to your doctor about whether EMPAVELI is right for you.
see what's possible with EMPAVELI
self-administration with the EMPAVELI Injector
connect with someone taking EMPAVELI
PNH=paroxysmal nocturnal hemoglobinuria .
Please see full Important Safety Information, including Boxed WARNING regarding risk of serious infections, on pages 38-39, and the full Prescribing Information and Medication Guide. EMPAVELI increases your chance of getting serious infections caused by encapsulated bacteria such as Streptococcus pneumoniae , Neisseria meningitidis , and Haemophilus influenzae type B. These serious infections may quickly become life-threatening or cause death if not recognized and treated early. 1. Y ou must complete or be up to date with the vaccines against Streptococcus pneumoniae and Neisseria meningitidis at least 2 weeks before your first dose of EMPAVELI. INDICATION AND IMPORTANT SAFETY INFORMATION What is EMPAVELI ® (pegcetacoplan)? EMPAVELI is a prescription medicine used to treat adults with a disease called paroxysmal nocturnal hemoglobinuria (PNH). What is the most important information I should know about EMPAVELI? EMPAVELI is a medicine that affects your immune system and may lower the ability of your immune system to fight infections.
Explore the compact, wearable EMPAVELI Injector! The EMPAVELI Injector allows you to self-administer your EMPAVELI when and where works best for you (after receiving proper training). Each EMPAVELI injection should take approximately 30 to 60 minutes.
Table of Contents
Hemolysis in PNH
4 6 8
How EMPAVELI works
take a look
Why EMPAVELI
How EMPAVELI can help
10
Possible side effects
26
Self-administration with the EMPAVELI Injector
28 32 35 36 38 40
[gl ow -bin] verb; Acting in pursuit of higher hemoglobin levels
ApellisAssist ® program
Helpful resources and tools
discover how you can be Globin’
PNH Peer 2 Peer program
Important Safety Information
Glossary
IMPORTANT SAFETY INFORMATION (cont’d) What is the most important information I should know about EMPAVELI? (cont'd) 2. If you have not completed your vaccines and EMPAVELI must be started right away, you should receive the required vaccines as soon as possible. 3. If you have not been vaccinated and EMPAVELI must be started right away, you should also receive antibiotics to take for as long as your healthcare provider tells you. 4. If you have been vaccinated against these bacteria in the past, you might need additional vaccines before starting EMPAVELI. Your healthcare provider will decide if you need additional vaccines.
PNH=paroxysmal nocturnal hemoglobinuria .
Please see full Important Safety Information, including Boxed WARNING regarding risk of serious infections, on pages 38-39, and the full Prescribing Information and Medication Guide.
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3
Hemolysis in PNH
With C5i treatments, some people with PNH may continue to have symptoms According to this survey, some people continued to have low Hb levels, ongoing fatigue, and the need for transfusions.
The impact of hemolysis in PNH Ongoing hemolysis may be causing your PNH symptoms
This could mean hemolysis is not under control.
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disease that involves the body’s immune system acting irregularly. In PNH, the immune system attacks and destroys its own red blood cells in a process called hemolysis. If hemolysis is not addressed, it can cause lower than normal levels of red blood cells and hemoglobin (Hb) in your blood. This may cause ongoing PNH symptoms, like fatigue, that force you to make compromises in your daily life.
In this survey, people reported:
Not achieving Hb levels ≥10.5 g/dL *
on eculizumab (20/32)
on ravulizumab (47/82)
Also, with anemia caused by low Hb levels, people with PNH may need to get frequent transfusions, which can take a toll on the body over time.
Ongoing fatigue †
on eculizumab (31/35)
on ravulizumab (65/87)
Ongoing need for transfusions ‡
on eculizumab (12/23)
on ravulizumab (7/31)
• 122 people taking a C5i treatment for their PNH were surveyed (35 received eculizumab, 87 received ravulizumab) • Most people surveyed (97%) had been taking C5i treatment for ≥3 months Possible limitations of the survey • A small number of people surveyed • People not satisfied with their current C5i treatment may have been more motivated to participate • Responses being subjective due to how people interpreted and reported their experiences • The results cannot be generalized to all people with PNH Findings from a US survey of people with PNH taking C5i treatment
C5i=C5 inhibitor.
‡ In patients who had a lifetime history of a transfusion and were on C5i treatment for at least 1 year. The need for transfusions was defined as 1 or more transfusions in the past 12 months. † Fatigue was measured using the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) scale, which is a type of survey that collects information about the impact of fatigue on different parts of daily life. The scale ranges from 0 to 52. Higher scores mean less fatigue. * Based on responses from 114 of the 122 survey participants who reported their Hb levels.
Don't delay! Talk to your healthcare provider about your ongoing PNH symptoms and available treatment options.
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5
How EMPAVELI works
How EMPAVELI works in PNH
EMPAVELI is the first and only C3i treatment for PNH that controls both types of hemolysis
EMPAVELI acts differently than other PNH treatments EMPAVELI works higher in the complement system than C5i treatments
intravascular hemolysis (IVH)
extravascular hemolysis (EVH)
The complement system is a part of your immune system and helps the body fight off infections. Certain proteins in the complement system work together and create a chain reaction. This chain reaction is called the complement cascade.
C3i=C3 inhibitor.
IMPORTANT SAFETY INFORMATION (cont’d) What is the most important information I should know about EMPAVELI? (cont'd) 5. Vaccines do not prevent all infections caused by encapsulated bacteria. Call your healthcare provider or get emergency medical care right away if you get any of these signs and symptoms of a serious infection: fever with or without shivers or the chills fever with chest pain and cough fever with high heart rate headache and a fever confusion clammy skin fever and a rash fever with breathlessness or fast breathing headache with nausea or vomiting headache with a stiff neck or stiff back body aches with flu-like symptoms eyes sensitive to light
PNH=paroxysmal nocturnal hemoglobinuria.
Please see full Important Safety Information, including Boxed WARNING regarding risk of serious infections, on pages 38-39, and the full Prescribing Information and Medication Guide.
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Why EMPAVELI
What is EMPAVELI? Discover what’s possible with EMPAVELI EMPAVELI is a prescription medicine used to treat adults with paroxysmal nocturnal hemoglobinuria (PNH).
EMPAVELI was studied in two Phase 3 clinical trials to find out how effective and safe EMPAVELI is in treating PNH:
Previous C5i treatment (PEGASUS)
No previous complement inhibitor treatment (PRINCE)
Compared EMPAVELI to a C5i treatment (eculizumab) in 80 adults with PNH who had previously received a C5i
Compared EMPAVELI to a control arm* (without EMPAVELI or any other complement inhibitor) in 53 adults with PNH who had never received a complement inhibitor
See how EMPAVELI was studied in PRINCE
See how EMPAVELI was studied in PEGASUS
*People in the control arm could continue taking stable doses of their medications for PNH except for EMPAVELI or any other complement inhibitors (like eculizumab or ravulizumab).
IMPORTANT SAFETY INFORMATION (cont’d) What is the most important information I should know about EMPAVELI? (cont'd) Your healthcare provider will give you a Patient Safety Card about the risk of serious infections. Carry it with you at all times during treatment and for 2 months after your last EMPAVELI dose. Your risk of serious infections may continue for several weeks after your last dose of EMPAVELI. It is important to show this card to any healthcare provider who treats you. This will help them diagnose and treat you quickly. EMPAVELI is only available through a program called the EMPAVELI Risk Evaluation and Mitigation Strategy (REMS). Before you can take EMPAVELI, your healthcare provider must enroll in the EMPAVELI REMS program, counsel you about the risk of serious infections caused by certain bacteria, give you information about the symptoms of serious infections, make sure that you are vaccinated against serious infections caused by encapsulated bacteria and that you receive antibiotics if you need to start EMPAVELI right away and you are not up to date on your vaccines, and give you a Patient Safety Card about your risk of serious infections.
Monica is an adult with PNH who's taken EMPAVELI.
Please see full Important Safety Information, including Boxed WARNING regarding risk of serious infections, on pages 38-39, and the full Prescribing Information and Medication Guide.
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How EMPAVELI can help | HEMOGLOBIN
Increased hemoglobin with EMPAVELI
Superior improvements in hemoglobin (Hb) in people who had previously received a C5i: results at Week 16 Primary goal in the PEGASUS study: to explore whether EMPAVELI was better at improving Hb levels than a C5i (eculizumab) in people living with paroxysmal nocturnal hemoglobinuria (PNH)
Peg is an adult with PNH who's taken EMPAVELI.
In people who had previously received a C5i (PEGASUS)
Hb improvement was maintained through the 48-week study
People taking EMPAVELI had a 2.37 g/dL Hb INCREASE
SUPERIOR Hb DIFFERENCE of 3.84 g/dL with EMPAVELI compared to eculizumab ( P <0.0001)
Average change in Hb levels from beginning to Week 48
People taking eculizumab had a 1.47 g/dL Hb DECREASE
View graph of Hb improvement over 16 weeks
IMPORTANT SAFETY INFORMATION (cont’d) Who should NOT take EMPAVELI? Do not take EMPAVELI if you: are allergic to pegcetacoplan or any of the ingredients in EMPAVELI.
C5i=C5 inhibitor; EMP=EMPAVELI; OLP=open-label period; RCP=randomized control period; SE=standard error.
have a serious infection caused by encapsulated bacteria, including Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B when you are starting EMPAVELI treatment.
Please see full Important Safety Information, including Boxed WARNING regarding risk of serious infections, on pages 38-39, and the full Prescribing Information and Medication Guide.
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How EMPAVELI can help | HEMOGLOBIN
Stabilized hemoglobin with EMPAVELI
Stabilized hemoglobin (Hb) levels in people who had never received a complement inhibitor: results at Week 26* One of the primary goals in the PRINCE study: to explore whether EMPAVELI stabilized hemoglobin in people living with PNH not previously treated with a complement inhibitor
In people who had never received a complement inhibitor (PRINCE)
Stacey is an adult with PNH who's taken EMPAVELI.
90
75
86% ( P <0.0001)
60
of people taking EMPAVELI achieved Hb stabilization over 26 weeks
45
30
15
0%
0
IMPORTANT SAFETY INFORMATION (cont’d) Before you take EMPAVELI, tell your healthcare provider about all of your medical
EMPAVELI n=35
Control arm (excluding complement inhibitors) n=18
conditions, including if you: have an infection or fever.
are pregnant or plan to become pregnant. EMPAVELI may harm your unborn baby. Females who are able to become pregnant should have a pregnancy test before starting treatment with EMPAVELI and use an effective method of birth control during treatment with EMPAVELI and for 40 days after the last dose. are breastfeeding or plan to breastfeed. It is not known if EMPAVELI passes into your breast milk. You should not breastfeed during treatment with EMPAVELI and for 40 days after the last dose.
*Hb stabilization means avoiding a >1 g/dL decrease in Hb levels throughout the study.
PNH=paroxysmal nocturnal hemoglobinuria.
Please see full Important Safety Information, including Boxed WARNING regarding risk of serious infections, on pages 38-39, and the full Prescribing Information and Medication Guide.
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How EMPAVELI can help | TRANSFUSIONS AND ARC
Avoiding the need for transfusions with EMPAVELI
Becoming transfusion-free with EMPAVELI vs eculizumab— in people who had previously received a C5i At Week 16 in PEGASUS, EMPAVELI was no worse than a C5i (eculizumab) in helping people with PNH become transfusion-free.
In people who had previously received a C5i (PEGASUS)
“I have not needed a transfusion since I switched to EMPAVELI.” Individual experiences may vary.
Jillian is an adult with PNH who's taken EMPAVELI.
of people taking EMPAVELI were transfusion-free
of people taking a C5i (eculizumab) were transfusion-free
Becoming transfusion-free with EMPAVELI vs control arm— in people who had never received a complement inhibitor At Week 26 in PRINCE, most people taking EMPAVELI were transfusion-free.
In people who had previously received a C5i (PEGASUS)
Average changes in ARC with EMPAVELI vs eculizumab— in people who had previously received a C5i People taking EMPAVELI saw their ARC reduced by 136 x 10 9 cells/L People taking a C5i (eculizumab) saw an increase in their ARC of 28 x 10 9 cells/L Reduced ARC At Week 16 in PEGASUS, EMPAVELI was no worse than a C5i (eculizumab) in helping people with PNH reduce their ARC.
In people who had never received a complement inhibitor (PRINCE)
of people taking EMPAVELI were transfusion-free
of people in the control arm (excluding complement inhibitors) were transfusion-free
IMPORTANT SAFETY INFORMATION (cont’d) Tell your healthcare provider about all the vaccines you receive and medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements which could affect your treatment.
ARC=absolute reticulocyte count; C5i=C5 inhibitor; PNH=paroxysmal nocturnal hemoglobinuria.
Please see full Important Safety Information, including Boxed WARNING regarding risk of serious infections, on pages 38-39, and the full Prescribing Information and Medication Guide.
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How EMPAVELI can help | LDH
Changes in LDH with EMPAVELI
Rapid and sustained LDH reductions with EMPAVELI vs control arm—in people who had never received a complement inhibitor One of the primary goals in the PRINCE study: to explore whether EMPAVELI reduced LDH in people living with PNH not previously treated with a complement inhibitor By Week 2 in PRINCE, average LDH levels in the EMPAVELI group had dropped from baseline and remained there through 26 weeks.
Louis is an adult with PNH who's taken EMPAVELI.
In people who had never received a complement inhibitor (PRINCE)
4000
3000
1535
2151
2000
1946
1000
400
205
(P <0.0001)
200
Normal range of LDH levels
0
Week 6
Week 12
Week 18
Week 26
34
33
33
34 30 7 5
EMPAVELI n Control arm n *
35 32 33 33 33 34 34 34 34 1817171713 9 8 8 8
7
8
6
Control arm (excluding complement inhibitors)
EMPAVELI
*Control arm (excluding complement inhibitors) only includes data on or before escape (switching to EMPAVELI).
At Week 26, average reductions in LDH levels were: -1870 U/L in the EMPAVELI group
-400 U/L in the control arm (excluding complement inhibitors)
IMPORTANT SAFETY INFORMATION (cont’d) If you stop taking EMPAVELI, your healthcare provider will need to monitor you closely for at least 8 weeks after stopping EMPAVELI. Stopping treatment with EMPAVELI may cause a breakdown of red blood cells due to PNH. Symptoms or problems that can happen due to red blood cell breakdown include: decreased hemoglobin level in your blood
Changes in LDH levels with EMPAVELI vs eculizumab— in people who had previously received a C5i (PEGASUS) At Week 16 in PEGASUS, it could not be determined if EMPAVELI was any worse than C5i (eculizumab) in reducing LDH.
tiredness pain in the stomach (abdomen) blood clots erectile dysfunction (ED)
C5i=C5 inhibitor; LDH=lactate dehydrogenase; PNH=paroxysmal nocturnal hemoglobinuria; SE=standard error.
blood in your urine shortness of breath trouble swallowing
Please see full Important Safety Information, including Boxed WARNING regarding risk of serious infections, on pages 38-39, and the full Prescribing Information and Medication Guide.
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How EMPAVELI can help | FATIGUE
Changes in fatigue
Changes in fatigue with EMPAVELI vs eculizumab—in people who had previously received a C5i (PEGASUS) Although it was not formally tested, people in the study were asked to report their changes in levels of fatigue at Week 16. Measuring fatigue improvement Fatigue was measured using the FACIT-Fatigue scale, a 13-question survey. In the survey, participants rated the level of impact they felt that their fatigue had on daily activities and function.
This information is for observation only and no conclusions can be made on the effect of EMPAVELI on fatigue. No comparisons can be made between those taking EMPAVELI and those taking eculizumab.
Average FACIT-Fatigue scores from beginning of study through Week 16
4-week run-in period (EMPAVELI + eculizumab)
16 weeks of EMPAVELI or eculizumab
20 25 30 35 40 45 50
This information is for observation only and no conclusions can be made on the effect of EMPAVELI on fatigue. No comparisons can be made between those taking EMPAVELI and those taking eculizumab.
43. 9
43.4
42. 2
42.5
43.6 average score for general US population
41. 8
41.0
40. 3
42. 8
32. 2
People taking EMPAVELI showed an improvement of
People taking C5i (eculizumab) showed a decrease of
32.7
32. 3
31.6
30. 8
30.7
30. 8
28.1
points
points
compared to their fatigue levels at the beginning of the study
compared to their fatigue levels at the beginning of the study
Week 2
Week 8
Baseline Day 1
Week 4 Week 6
Week 12
Week 16
EMPAVELI n eculizumab n 38 41
37 41
38 40
37 39
39 39
38 38
39 38
38 36
EMPAVELI
eculizumab
An increase of 5 or more points is seen as a clinically meaningful improvement.
The FACIT-Fatigue scale ranges from 0 to 52. Higher scores mean less fatigue. These data include people who did and did not get a transfusion during the study.
IMPORTANT SAFETY INFORMATION (cont’d) What are the possible side effects of EMPAVELI?
Andrea is an adult with PNH who's taken EMPAVELI.
EMPAVELI can cause serious side effects including allergic reactions. Allergic reactions can happen during your EMPAVELI infusion. Stop your EMPAVELI infusion and tell your healthcare provider or get emergency medical care right away if you get any of these symptoms during your EMPAVELI infusion: chest pain
Please see full Important Safety Information, including Boxed WARNING regarding risk of serious infections, on pages 38-39, and the full Prescribing Information and Medication Guide. C5i=C5 inhibitor; FACIT=Functional Assessment of Chronic Illness Therapy; PNH=paroxysmal nocturnal hemoglobinuria; SE=standard error.
trouble breathing or shortness of breath swelling of your face, tongue, or throat feel faint or pass out
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How EMPAVELI can help | FATIGUE
A post hoc analysis of PEGASUS data found a link between Hb and fatigue
“I love leisurely walks and vacations with my family. With my increase in Hb levels and energy, I don’t have to miss out. ” Individual experiences may vary.
In people who had previously received a C5i (PEGASUS)
10 12 14
2 4 6 8
Andrea is an adult with PNH who's taken EMPAVELI.
People with greater improvements in Hb showed larger reductions in fatigue
11.3
-1.2
9.9
0 -2 -4
<1.0 g/dL n=39
Increase in Hb ≥1.0-<2.0 g/dL n=8
≥2.0 g/dL n=25
People with PNH who had an Hb improvement of 1 g/dL or more had an average fatigue improvement of ~10 points .
A post hoc analysis gathers additional findings after a study has ended. It is not considered as scientifically strong as an analysis that was planned before the study began This analysis included people receiving EMPAVELI and people receiving eculizumab C5i=C5 inhibitor; FACIT=Functional Assessment of Chronic Illness Therapy; Hb=hemoglobin; PNH=paroxysmal nocturnal hemoglobinuria. Please see full Important Safety Information, including Boxed WARNING regarding risk of serious infections, on pages 38-39, and the full Prescribing Information and Medication Guide.
IMPORTANT SAFETY INFORMATION (cont’d) What are the possible side effects of EMPAVELI? (cont'd)
The most common side effects in people with PNH treated with EMPAVELI include injection-site reactions; infections; diarrhea; pain in the stomach (abdomen); respiratory tract infection; pain in the arms, hands, legs, or feet; low potassium in blood; tiredness; viral infection; cough; joint pain; dizziness; headache; and rash.
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How EMPAVELI can help | Hb NORMALIZATION
Getting to normalization of lab values What is normalization? How is it different than stabilization? Normalization means lab values improved to levels like those of adults who don’t have PNH. This is different than stabilization, which generally means that lab values did not get worse. Hemoglobin (Hb) normalization in people with and without previous complement inhibitor treatment This information is for observation only and no conclusions can be made on the effect of EMPAVELI on Hb normalization. No comparisons can be made between those taking EMPAVELI and those taking eculizumab in PEGASUS or those in the control arm (excluding complement inhibitors) in PRINCE.
Louis is an adult with PNH who's taken EMPAVELI.
In people who had never received a complement inhibitor (PRINCE)
In people who had previously received a C5i (PEGASUS)
At Week 26 in PRINCE
At Week 16 in PEGASUS
60
40
35
50
34%
30
46%
of people taking EMPAVELI achieved Hb normalization
of people taking EMPAVELI achieved Hb normalization
40
25
30
20
15
20
Hb normalization means their Hb got to within the normal range (12-16 g/dL for females, 13.6-18 g/dL for males).
Hb normalization means their Hb got to within the normal range (12-16 g/dL for females, 13.6-18 g/dL for males).
10
10
5
0%
0%
0
0
Control arm (excluding complement inhibitors) n=18
EMPAVELI n=35
EMPAVELI n=41
eculizumab n=39
These data include only people who did not require a transfusion during the study.
These data include only people who did not require a transfusion during the study.
IMPORTANT SAFETY INFORMATION (cont’d) What are the possible side effects of EMPAVELI? (cont'd) These are not all of the possible side effects of EMPAVELI. Tell your healthcare provider about any side effect that bothers you or that does not go away. Call your healthcare provider for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
C5i=C5 inhibitor; PNH=paroxysmal nocturnal hemoglobinuria.
Please see full Important Safety Information, including Boxed WARNING regarding risk of serious infections, on pages 38-39, and the full Prescribing Information and Medication Guide.
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How EMPAVELI can help | LDH NORMALIZATION
The majority of people taking EMPAVELI reduced their LDH levels to within the normal range (113-226 U/L). This information is for observation only and no conclusions can be made on the effect of EMPAVELI on LDH normalization. No comparisons can be made between those taking EMPAVELI and those taking eculizumab in PEGASUS or those in the control arm (excluding complement inhibitors) in PRINCE. LDH normalization in people with and without previous complement inhibitor treatment
Nearly 2/3 of people taking EMPAVELI reduced their LDH levels to within the normal range (113-226 U/L).
Jillian is an adult with PNH who's taken EMPAVELI.
In people who had never received a complement inhibitor (PRINCE)
In people who had previously received a C5i (PEGASUS)
At Week 26 in PRINCE
80
At Week 16 in PEGASUS
80
70
70
60
66%
71%
of people taking EMPAVELI achieved LDH normalization
of people taking EMPAVELI achieved LDH normalization
60
50
50
40
40
30
30
20
20
10
0%
15%
10
0
Control arm (excluding complement inhibitors) n=18
EMPAVELI n=35
0
EMPAVELI n=41
eculizumab n=39
These data include only people who did not require a transfusion during the study.
These data include only people who did not require a transfusion during the study.
Long-term Hb and LDH data
see additional data
IMPORTANT SAFETY INFORMATION What is the most important information I should know about EMPAVELI? EMPAVELI is a medicine that affects your immune system and may lower the ability of your immune system to fight infections.
C5i=C5 inhibitor; Hb=hemoglobin; LDH=lactate dehydrogenase; PNH=paroxysmal nocturnal hemoglobinuria.
Please see full Important Safety Information, including Boxed WARNING regarding risk of serious infections, on pages 38-39, and the full Prescribing Information and Medication Guide.
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Possible side effects
Most common side effects
Infections
Abdominal pain
Injection site reactions
Diarrhea
Respiratory tract infection Viral infection
Tiredness
EMPAVELI is only available through a program called the EMPAVELI REMS (Risk Evaluation and Mitigation Strategy). REMS is a safety program run by the FDA. Before you can take EMPAVELI, your healthcare provider must enroll in the program and will provide you with the following: Counseling on the risk of serious infections caused by certain bacteria Information about the symptoms of serious infections Appropriate vaccinations against serious infections caused by encapsulated bacteria – You will receive antibiotics if you need to start EMPAVELI right away and are not up to date on your vaccines A Patient Safety Card – Carry this card with you at all times during treatment and for 2 months after your last EMPAVELI dose – Show this card to any healthcare professional to help diagnose and treat you quickly – Your risk of serious infection may continue for several weeks after your last dose of EMPAVELI EMPAVELI REMS program ensures your safety is a top priority
Serious adverse reactions were reported in 17% of patients treated with EMPAVELI - T he most common serious adverse events were infections (5%) Injection/infusion site reactions were mild or moderate in severity. Seventeen cases of diarrhea were reported during the 48 weeks. Fifteen of the cases were mild and 2 were moderate Breakthrough hemolysis occurred in 10% of those taking EMPAVELI vs 23% of those taking eculizumab at Week 16 in the PEGASUS study. Three people taking EMPAVELI left the study due to hemolysis
Injection site reactions
Infections
Viral infection
Pain in extremity
Low potassium
Joint pain
Dizziness
Abdominal pain
Rash
Headache
One patient treated with EMPAVELI died due to septic shock Serious adverse reactions were reported in 13% of patients treated with EMPAVELI Overall, there were no EMPAVELI-related side effects leading to drug or trial discontinuation No events of acute hemolysis were observed in either treatment group through Week 26 These are not all the possible side effects of EMPAVELI. Be sure to tell your healthcare provider about any side effect that bothers you or that does not go away. Side effects can be reported to FDA at 1-800-FDA-1088 or www.fda.gov/medwatch Two people taking EMPAVELI in the PEGASUS and PRINCE studies had side effects related to blood clots. They were deemed not related to EMPAVELI There have been no cases of meningococcal infections in any adults with PNH treated with pegcetacoplan, as of 11/13/2023. They were vaccinated prior to treatment with pegcetacoplan
Please see full Important Safety Information, including Boxed WARNING regarding risk of serious infections, on pages 38-39, and the full Prescribing Information and Medication Guide.
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Self-administration with the EMPAVELI Injector
Self-administration with the
About the EMPAVELI Injector
4 Push button starts injection and pops up when injection is complete
EMPAVELI self-administration may fit into your lifestyle EMPAVELI is a PNH treatment that you give yourself at home or wherever works for you (after receiving proper training). EMPAVELI is self-administered subcutaneously (just under the skin). Self-administering EMPAVELI should take approximately 30 to 60 minutes.
4 The needle is never seen
4 Compact device with no tubing involved
No intravenous (IV) infusions
4 The gauge shows the injection progress
No infusion center visits
No need to plan around infusion appointments
You will be trained before using the EMPAVELI Injector for the first time. Refer to the EMPAVELI Injector Instructions for Use for more information.
EMPAVELI can be self-administered using either: The EMPAVELI Injector; or A commercially available infusion pump with a reservoir of at least 20 mL
“I love to travel. With EMPAVELI self-administration, I can bring my supplies with me and travel without
PNH=paroxysmal nocturnal hemoglobinuria.
interrupting my treatment schedule.” Emma is an adult with PNH who's taken EMPAVELI. Individual experiences may vary.
IMPORTANT SAFETY INFORMATION (cont’d) What is the most important information I should know about EMPAVELI? (cont’d) EMPAVELI increases your chance of getting serious infections caused by encapsulated bacteria such as Streptococcus pneumoniae , Neisseria meningitidis , and Haemophilus influenzae type B. These serious infections may quickly become life-threatening or cause death if not recognized and treated early. 1. Y ou must complete or be up to date with the vaccines against Streptococcus pneumoniae and Neisseria meningitidis at least 2 weeks before your first dose of EMPAVELI.
When traveling, ensure that EMPAVELI stays at 36°F to 46°F (2°C-8°C).
Please see full Important Safety Information, including Boxed WARNING regarding risk of serious infections, on pages 38-39, and the full Prescribing Information and Medication Guide.
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Self-administration with the EMPAVELI Injector
How do I take EMPAVELI? EMPAVELI is self-administered just under the skin using a small, thin needle that you never see with the EMPAVELI Injector. EMPAVELI is not an IV infusion (into the vein) that requires a healthcare provider to administer it for you. How often do I take EMPAVELI? EMPAVELI is taken 2 times a week* The injection takes approximately 30 to 60 minutes
See the EMPAVELI Injector Instructions for Use or, if using an infusion pump, see these specific Instructions for Use .
Avoid intense physical activity and do not bump or knock the EMPAVELI Injector or button during the injection. Keep your stomach totally dry.
If you miss a dose of EMPAVELI, take the missed dose as soon as possible. Take your next dose as regularly planned.
For ~30-60 minutes
*If there is an increase in your LDH, an enzyme in your blood, your healthcare provider may tell you to take EMPAVELI every 3 days.
If your healthcare provider decides to stop your treatment with EMPAVELI, follow their instructions for how to stop. Your healthcare provider will monitor you closely for at least 8 weeks after stopping treatment with EMPAVELI for any signs of hemolysis due to PNH.
watch the EMPAVELI Injector self-administration video
you get self-administration support from the start
there are processes for transitioning to EMPAVELI
certain vaccines are required before starting EMPAVELI
LDH=lactate dehydrogenase; PNH=paroxysmal nocturnal hemoglobinuria.
IMPORTANT SAFETY INFORMATION (cont’d) What is the most important information I should know about EMPAVELI? (cont'd) 2. If you have not completed your vaccines and EMPAVELI must be started right away, you should receive the required vaccines as soon as possible. 3. If you have not been vaccinated and EMPAVELI must be started right away, you should also receive antibiotics to take for as long as your healthcare provider tells you. 4. If you have been vaccinated against these bacteria in the past, you might need additional vaccines before starting EMPAVELI. Your healthcare provider will decide if you need additional vaccines.
Please see full Important Safety Information, including Boxed WARNING regarding risk of serious infections, on pages 38-39, and the full Prescribing Information and Medication Guide.
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ApellisAssist ® program
Emma is an adult with PNH who's taken EMPAVELI.
“My ACE is amazing. Her training helped give me confidence in self-administering EMPAVELI. She continues to be there for me and is always a phone call away.” Individual experiences may vary.
Support every step of the way
The ApellisAssist program is here for you
It takes teamwork to help manage PNH. The ApellisAssist patient support program provides comprehensive support, with helpful resources and experienced people dedicated to making a difference for you. With the program, you have access to: Financial assistance (for those who are eligible) Insurance support Self-administration training from an Apellis Care Educator (ACE) EMPAVELI and PNH education
Your ApellisAssist support partners With EMPAVELI, you don’t have to go it alone. Learn about your dedicated partners from the ApellisAssist program.
Care Coordinator Through our specialty pharmacy, PANTHERx Rare, a Care Coordinator will: Help schedule your deliveries for medication and supplies Explain your insurance benefits or available financial assistance options, if eligible
Apellis Care Educator (ACE) Your ACE is there for you from the start. They will provide you: 1-on-1 training on how to self-administer EMPAVELI—in your home or virtually Ongoing support every step of the way A wide variety of educational resources Answers to questions that may pop up
1-866-MY-APL-ASSIST (1-866-692-7527) FROM 8 am -8 pm ET, MONDAY-FRIDAY
Care Coordinators and ACEs do not give medical advice. Talk to your doctor for treatment-related questions.
PNH=paroxysmal nocturnal hemoglobinuria.
Please see full Important Safety Information, including Boxed WARNING regarding risk of serious infections, on pages 38-39, and the full Prescribing Information and Medication Guide.
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ApellisAssist ® program
Helpful resources and tools
Tools for your journey Resources, tools, and tips designed for you No matter where you are in your journey, there are many resources available to help.
8 weeks of EMPAVELI treatment at no cost to eligible adults with PNH* If you haven’t tried EMPAVELI yet, you may be eligible for the EMPAVELI Trial Offer regardless of insurance coverage or current treatment.
A range of brochures and guides available to download
Straightforward enrollment for you and your healthcare provider
The same comprehensive training and support as those currently taking EMPAVELI
Extensive video content including patient stories, ACE support stories, and step-by-step instructions for taking EMPAVELI
No obligation to continue with EMPAVELI after trial ends
Home Suite Home with tools and videos for traveling with, storing, and organizing your medication
If you and your healthcare provider are ready to try EMPAVELI, you may be eligible to enroll in the Trial Offer program .
*Terms, conditions, and eligibility requirements apply. Ask your doctor for more information.
Social communities to keep in touch on Facebook, Instagram, and YouTube
Insurance questions? In need of financial assistance?
Call an ApellisAssist representative at 1-866-MY-APL-ASSIST (1-866-692-7527) to connect with your Care Coordinator about insurance questions or financial assistance programs that are available for eligible patients.
Angelo is an adult with PNH who's taken EMPAVELI.
access these helpful resources for your EMPAVELI journey
Please see full Important Safety Information, including Boxed WARNING regarding risk of serious infections, on pages 38-39, and the full Prescribing Information and Medication Guide.
ACE=Apellis Care Educator; PNH=paroxysmal nocturnal hemoglobinuria.
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PNH Peer 2 Peer program
A loving grandmother who remained resilient in her path to PNH care Andrea
A lover of the outdoors who found courage in the face of the unknown Stacey
Connect 1-on-1 with someone taking EMPAVELI . The PNH Peer 2 Peer program gives you the chance to connect directly with someone else who is living with PNH.
Hear about their journey living with PNH, ask them questions, and find out about their experiences starting or switching to EMPAVELI.
Your healthcare provider is your best source of information regarding your PNH.
Andrea and Stacey are adults with PNH who've taken EMPAVELI.
meet a Peer 2 Peer Connector! learn about our Connectors and schedule a time to meet
Stacey is an adult with PNH who's taken EMPAVELI.
PNH=paroxysmal nocturnal hemoglobinuria.
Please see full Important Safety Information, including Boxed WARNING regarding risk of serious infections, on pages 38-39, and the full Prescribing Information and Medication Guide.
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INDICATION AND IMPORTANT SAFETY INFORMATION
Who should NOT take EMPAVELI? Do not take EMPAVELI if you: are allergic to pegcetacoplan or any of the ingredients in EMPAVELI.
What is EMPAVELI ® (pegcetacoplan)? EMPAVELI is a prescription medicine used to treat adults with a disease called paroxysmal nocturnal hemoglobinuria (PNH). What is the most important information I should know about EMPAVELI? EMPAVELI is a medicine that affects your immune system and may lower the ability of your immune system to fight infections. EMPAVELI increases your chance of getting serious infections caused by encapsulated bacteria such as Streptococcus pneumoniae , Neisseria meningitidis , and Haemophilus influenzae type B. These serious infections may quickly become life-threatening or cause death if not recognized and treated early. 1. Y ou must complete or be up to date with the vaccines against Streptococcus pneumoniae and Neisseria meningitidis at least 2 weeks before your first dose of EMPAVELI. 2. If you have not completed your vaccines and EMPAVELI must be started right away, you should receive the required vaccines as soon as possible. 3. If you have not been vaccinated and EMPAVELI must be started right away, you should also receive antibiotics to take for as long as your healthcare provider tells you. 4. If you have been vaccinated against these bacteria in the past, you might need additional vaccines before starting EMPAVELI. Your healthcare provider will decide if you need additional vaccines. 5. Vaccines do not prevent all infections caused by encapsulated bacteria. Call your healthcare provider or get emergency medical care right away if you get any of these signs and symptoms of a serious infection: fever with or without shivers or the chills fever with chest pain and cough fever with high heart rate headache and a fever confusion clammy skin fever and a rash fever with breathlessness or fast breathing headache with nausea or vomiting headache with a stiff neck or stiff back body aches with flu-like symptoms eyes sensitive to light Your healthcare provider will give you a Patient Safety Card about the risk of serious infections. Carry it with you at all times during treatment and for 2 months after your last EMPAVELI dose. Your risk of serious infections may continue for several weeks after your last dose of EMPAVELI. It is important to show this card to any healthcare provider who treats you. This will help them diagnose and treat you quickly. EMPAVELI is only available through a program called the EMPAVELI Risk Evaluation and Mitigation Strategy (REMS). Before you can take EMPAVELI, your healthcare provider must enroll in the EMPAVELI REMS program, counsel you about the risk of serious infections caused by certain bacteria, give you information about the symptoms of serious infections, make sure that you are vaccinated against serious infections caused by encapsulated bacteria and that you receive antibiotics if you need to start EMPAVELI right away and you are not up to date on your vaccines, and give you a Patient Safety Card about your risk of serious infections.
have a serious infection caused by encapsulated bacteria, including Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B when you are starting EMPAVELI treatment. Before you take EMPAVELI, tell your healthcare provider about all of your medical conditions, including if you: have an infection or fever. are pregnant or plan to become pregnant. EMPAVELI may harm your unborn baby. Females who are able to become pregnant should have a pregnancy test before starting treatment with EMPAVELI and use an effective method of birth control during treatment with EMPAVELI and for 40 days after the last dose. are breastfeeding or plan to breastfeed. It is not known if EMPAVELI passes into your breast milk. You should not breastfeed during treatment with EMPAVELI and for 40 days after the last dose. Tell your healthcare provider about all the vaccines you receive and medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements which could affect your treatment. If you stop taking EMPAVELI, your healthcare provider will need to monitor you closely for at least 8 weeks after stopping EMPAVELI. Stopping treatment with EMPAVELI may cause a breakdown of red blood cells due to PNH. Symptoms or problems that can happen due to red blood cell breakdown include: decreased hemoglobin level in your blood
tiredness pain in the stomach (abdomen) blood clots erectile dysfunction (ED)D)
blood in your urine shortness of breath trouble swallowing
What are the possible side effects of EMPAVELI? EMPAVELI can cause serious side effects including allergic reactions. Allergic reactions can happen during your EMPAVELI infusion. Stop your EMPAVELI infusion and tell your healthcare provider or get emergency medical care right away if you get any of these symptoms during your EMPAVELI infusion: chest pain
trouble breathing or shortness of breath swelling of your face, tongue, or throat feel faint or pass out
The most common side effects in people with PNH treated with EMPAVELI include injection-site reactions; infections; diarrhea; pain in the stomach (abdomen); respiratory tract infection; pain in the arms, hands, legs, or feet; low potassium in blood; tiredness; viral infection; cough; joint pain; dizziness; headache; and rash. These are not all of the possible side effects of EMPAVELI. Tell your healthcare provider about any side effect that bothers you or that does not go away. Call your healthcare provider for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. Please see full Prescribing Information, including Boxed WARNING regarding risk of serious infections, and Medication Guide for additional information.
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Absolute reticulocyte count (ARC) A test that measures the number of immature red blood cells in your blood. High reticulocyte counts may be a sign of ongoing hemolysis with paroxysmal nocturnal hemoglobinuria (PNH), as the body is making more new red blood cells to replace the ones being destroyed by the immune system. C3 A protein in the blood that acts as a central point of the complement system, regulating its activation. C5 Another protein in the blood that is part of the complement system. Clinical trial A type of research study that tests how well treatments work in people, as well as possible side effects of treatments. Complement system The complement system, also referred to as the complement cascade, is an army of more than 40 different proteins that work together to get rid of dangerous cells and fight infection.
Hemolysis The breaking apart of red blood cells. It can occur when the immune system attacks these cells as though they were dangerous viruses or bacteria. When red blood cells break open, hemoglobin is released. Hemolysis causes many of the symptoms of PNH. Immune system A sophisticated defense network used to protect the body from dangers like disease and infection.
Intravascular hemolysis (IVH) The destruction of red blood cells inside a blood vessel.
Lactate dehydrogenase (LDH) An enzyme found in the blood and tissues of the body, including the heart, kidneys, brain, and lungs. Red blood cell destruction results in the release of LDH into the blood. People with PNH often have higher levels of LDH due to ongoing hemolysis.
Normalization Lab values improving to levels like those of adults who don’t have PNH.
Proteins Molecules that play many important roles in the body and can be thought of as the “workhorses” of cells. Proteins are required for the structure, function, and regulation of the body’s organs and tissues. Red blood cells A common type of blood cell. Their job is to carry oxygen, using an important molecule, hemoglobin, around the body.
Extravascular hemolysis (EVH) Destruction of red blood cells that occurs in the liver or spleen.
FACIT-Fatigue scale This tool consists of a number of questions that are used to assign a score to measure a person’s experience with fatigue.
Stabilization Lab values remaining at a stable level and not getting worse.
Globin' Acting in pursuit of higher hemoglobin levels.
U/L Some test results are reported in U/L, or units per liter. This is a measurement of how many units there are in a liter. x 10 9 cells/L A short way to write a number with 9 zeros at the end. This is useful when needing to count the billions of cells in a liter of blood.
Hemoglobin (Hb) The critical protein found inside red blood cells that enables other cells throughout the body to get the oxygen they need. Hemoglobin acts like drops of glue that can “stick” to oxygen and carry it from the lungs to other tissues. It can also “stick” to waste like carbon dioxide to help remove it from the body.
Please see full Important Safety Information, including Boxed WARNING regarding risk of serious infections, on pages 38-39, and the full Prescribing Information and Medication Guide.
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it’s time to get Globin’ discover more about what's possible with EMPAVELI
If you're like Monica, an adult with PNH taking EMPAVELI,
Where will you go next?
explore the EMPAVELI Injector get details and resources on using the EMPAVELI Injector
you don’t have to go it alone your ApellisAssist team, including your ACE, is always at your side
learn more
see how
ACE=Apellis Care Educator; PNH=paroxysmal nocturnal hemoglobinuria.
Please see full Important Safety Information, including Boxed WARNING regarding risk of serious infections, on pages 38-39, and the full Prescribing Information and Medication Guide. EMPAVELI increases your chance of getting serious infections caused by encapsulated bacteria such as Streptococcus pneumoniae , Neisseria meningitidis , and Haemophilus influenzae type B. These serious infections may quickly become life-threatening or cause death if not recognized and treated early. 1. Y ou must complete or be up to date with the vaccines against Streptococcus pneumoniae and Neisseria meningitidis at least 2 weeks before your first dose of EMPAVELI. INDICATION AND IMPORTANT SAFETY INFORMATION What is EMPAVELI ® (pegcetacoplan)? EMPAVELI is a prescription medicine used to treat adults with a disease called paroxysmal nocturnal hemoglobinuria (PNH). What is the most important information I should know about EMPAVELI? EMPAVELI is a medicine that affects your immune system and may lower the ability of your immune system to fight infections.
APELLIS, APELLISASSIST, EMPAVELI, and their respective logos are registered trademarks of Apellis Pharmaceuticals, Inc. Other trademarks referenced herein are the property of their respective owners. ©2023-2024 Apellis Pharmaceuticals, Inc. All rights reserved. 5/24 US-PEGPNH-2300265 v3.0
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