“with empaveli, my hemoglobin is higher

POSSIBILITIES IN PNH

What is EMPAVELI?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 can affect your immune system and can lower the ability of your immune system to fight infections.�EMPAVELI may increase your chance of getting serious and life‑threatening meningococcal infections that may quickly become life‑threatening and cause death if not recognized and treated early.�EMPAVELI may also increase the risk of getting serious infections caused by certain bacteria such as Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B. Serious infections may quickly become life‑threatening and cause death if not recognized and treated early. – You must be vaccinated against these bacteria at least 2 weeks before your first dose of EMPAVELI if you have not already had these vaccines.

Please see Important Safety Information, including Boxed WARNING regarding risk of serious infections, on pages 23-25, full Prescribing Information, and Medication Guide.

EMPAVELI was studied vs eculizumab in a head‑to‑head Phase 3 clinical trial in 80 people living with PNH. Compared to the beginning of the study, people taking EMPAVELI had a 2.37 g/dL increase in hemoglobin levels compared to a 1.47 g/dL decrease with eculizumab at Week 16. EMPAVELI showed a superior 3.84 g/dL difference in Hb levels vs eculizumab (P<0.0001).

“With EMPAVELI, my hemoglobin is higher.

I feel really good about that, and my doctor agrees.”

Image is an actor portrayal.Quote from a real EMPAVELI patient.Talk to your doctor about whether

EMPAVELI is right for you. Image is an actor portrayal.

https://pi.apellis.com/files/PI_Empaveli.pdf

https://pi.apellis.com/files/MedGuide_Empaveli.pdf

2

Talk to your doctor to discover what EMPAVELI could mean for you


Table of ContentsWhy hemolysis matters in PNH 4

How EMPAVELI works 7

How was EMPAVELI studied? 8

Important information about EMPAVELI 15

How to take EMPAVELI 19

ApellisAssist™ Support 21

Important Safety Information 23

For definitions of terms used throughout this brochure, refer to the glossary, which can be found in the back of this brochure.



Image is an actor portrayal.

Paroxysmal nocturnal hemoglobinuria (PNH) is a blood disease in which the immune system—the body’s natural defense system— attacks and destroys red blood cells.




4

Are you still experiencing PNH symptoms? They may be the result of ongoing hemolysis

What is hemolysis?

is the destruction of red blood cells by the complement system

HEMOLYSIS

Despite improvements while on treatment, some people living with PNH still experience:

�Fatigue

�Abnormal lab values

These may be signs that your red blood cells are being destroyed in the body.

There are 2 types of hemolysis in PNH Most treatments only help with 1 kind of hemolysis, known as intravascular hemolysis

What are the 2 types of hemolysis?

is when red blood cells are destroyed inside the blood vessels

INTRAVASCULAR HEMOLYSIS (IVH)

is when red blood cells are destroyed outside the blood vessels, in the liver and spleen

EXTRAVASCULAR HEMOLYSIS (EVH)

Both types of hemolysis may be responsible for ongoing PNH symptoms

5


EMPAVELI™ (pegcetacoplan) is the first and only FDA-approved

PNH medication that:

CONTROLS BOTH FORMS OF

HEMOLYSIS— IVH & EVH —


IMPORTANT SAFETY INFORMATION (cont’d)What is the most important information I should know about EMPAVELI? (cont’d) If you have been vaccinated against these bacteria in the past, you might need additional vaccinations before starting EMPAVELI. Your healthcare provider will decide if you need additional vaccinations. Vaccines reduce the risk of serious infections, but do not prevent all serious infections. 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 and a rash�shortness of breath�extreme pain or discomfort

�headache with nausea or vomiting�high heart rate�headache and a fever �headache with a stiff neck or stiff back

�confusion�muscle aches with flu-like symptoms�clammy skin�eyes sensitive to light



7


C3

C3a C3b

C5

C5a C5b

How EMPAVELI worksIt works higher in the complement system than other treatments, like eculizumab

The complement system is a part of your immune system and helps your body fight off infections. In the complement system, proteins work together, creating a chain reaction.

This chain reaction is called the complement cascade.

EMPAVELI is the first and only treatment for PNH that targets C3 and may prevent both forms of hemolysis

EMPAVELI prevents C3 from splitting into C3a and C3b, helping to stop IVH and EVH

C5 treatments, like eculizumab, work on C5

C3 is the central protein in the chain reaction

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 infection. Carry it with you at all times during treatment and for 2 months after your last EMPAVELI dose. It is important to show this card to any healthcare provider to help them diagnose and treat you quickly.



8

How was EMPAVELI studied?

People in this trial needed to have been on a steady dose of eculizumab for at least 3 months and have low hemoglobin (Hb) levels (less than 10.5 g/dL) in order to be eligible for the study.

EMPAVELI was studied in a head-to-head, 16-week, open-label clinical trial compared to eculizumab in 80 people living with PNH.

About the studyThe study consisted of 2 parts:

�A 4-week period in which people received both EMPAVELI and eculizumab

�After 4 weeks, people were randomly split into 2 groups, in which they received only EMPAVELI or only eculizumab for 16 weeks

Goals of the study included:

�Primary goal: to explore changes in Hb levels

�Secondary goal: to study additional data points

�Safety: to monitor the occurrence of side effects

Please see the following pages for more details on the above goals and study results.


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.�have not been vaccinated against Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B, unless your healthcare provider decides that urgent treatment with EMPAVELI is needed.�have a serious infection caused by the bacteria above.



9

Superior improvements in Hb

+2.37in Hb levels with EMPAVELI

GRAM/dLINCREASE

–1.47in Hb levels with eculizumab

GRAM/dL DECREASE

Compared to the beginning of the study, people taking either treatment had the following at Week 16:

PRIMARY GOAL: To explore whether EMPAVELI was better at improving Hb levels than eculizumab in people living with PNH.

3.84in Hb levels vs eculizumab (P<0.0001)

EMPAVELI showed a superior


IMPORTANT SAFETY INFORMATION (cont’d)

Before you take EMPAVELI, tell your healthcare provider about all 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 final 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 final dose.

GRAM/dL DIFFERENCE



10

KEY SECONDARY GOAL:

“Noninferiority” tests were used to study additional information and determine if EMPAVELI was no worse than eculizumab compared to the beginning of the study. These include:

�Helping people become transfusion free

� Changes in reticulocytes (also known as absolute reticulocyte count, or ARC)

�Changes in LDH (also known as lactate dehydrogenase)

�Changes in fatigue

Fatigue was measured using a survey called Functional Assessment of Chronic Illness Therapy (FACIT-Fatigue Scale), where higher scores on the survey meant a person felt less fatigue.

LDH was tested for and did not meet noninferiority, but FACIT-Fatigue was not tested. This information was for observation only.

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:

Additional values studied


�decreased hemoglobin level in your blood�blood in your urine�shortness of breath�trouble swallowing

�tiredness�pain in the stomach (abdomen)�blood clots�erectile dysfunction (ED)



11

In this study, EMPAVELI was no worse than eculizumab in achieving transfusion independence and reducing ARC.At Week 16:

EMPAVELI impacts these values that may play a role in PNH


Additional values studied (cont’d)

IMPORTANT SAFETY INFORMATION (cont’d) 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:

Transfusion independence was acheived for 35 people(85%) taking EMPAVELI who were transfusion free

Reduced ARC for people taking EMPAVELI by 136 X 109 cells/L compared to their values at the beginning of the study

�chest pain�trouble breathing or shortness of breath

�swelling of your face, tongue, or throat�feel faint or pass out



12

LDH was tested for noninferiority, but it could not be determined if EMPAVELI was no worse than eculizumab. Information collected was for observation only and no conclusions can be made on the effect of EMPAVELI on this measure.

�At Week 16, people taking EMPAVELI had reduced LDH by 15 U/L compared to their LDH values at the beginning of the study

FACIT-Fatigue was not formally tested. Information collected was for observation only and no conclusions can be made on the effect of EMPAVELI on this measure.

�At Week 16, people taking EMPAVELI had improved fatigue by 9.2 points on the FACIT-Fatigue scale compared to their fatigue levels at the beginning of the study

Additional results from the study


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, viral infection, and tiredness.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.



13

Normalization of lab valuesHb and ARC normalization were prespecified secondary endpoints, or goals planned to be evaluated prior to the start of the PEGASUS study

Information collected was for observation only. No conclusions can be made from these data.

In this analysis of prespecified secondary endpoints in the PEGASUS study, at Week 16:

�Hb normalization was achieved in 34% of people taking EMPAVELI

Normal Hb is usually between 12 g/dL and 18 g/dL, depending on sex.

�ARC normalization was achieved in 78% of people taking EMPAVELI

Normal ARC can differ from person to person. It is measured as a range of values, from 30 X 109 to 120 X 109 cells/L.

IMPORTANT SAFETY INFORMATION (cont’d) What is the most important information I should know about EMPAVELI?EMPAVELI is a medicine that can affect your immune system and can lower the ability of your immune system to fight infections.�EMPAVELI may increase your chance of getting serious and life‑threatening meningococcal infections that may quickly become life‑threatening and cause death if not recognized and treated early.�EMPAVELI may also increase the risk of getting serious infections caused by certain bacteria such as Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B. Serious infections may quickly become life‑threatening and cause death if not recognized and treated early. – You must be vaccinated against these bacteria at least 2 weeks before your first dose of EMPAVELI if you have not already had these vaccines.




24 people living with PNH were studied in 2 uncontrolled clinical trials. EMPAVELI was not compared against another treatment. These patients had never received complement treatment before and had at least 1 transfusion in the last year before entering the trial

Patients were on EMPAVELI for about 1 year and increases in hemoglobin were seen

EMPAVELI was also studied in patients who have never received PNH treatment with a complement inhibitor before

14


IMPORTANT SAFETY INFORMATION (cont’d)What is the most important information I should know about EMPAVELI? (cont’d) If you have been vaccinated against these bacteria in the past, you might need additional vaccinations before starting EMPAVELI. Your healthcare provider will decide if you need additional vaccinations. Vaccines reduce the risk of serious infections, but do not prevent all serious infections. 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 and a rash�shortness of breath�extreme pain or discomfort

�headache with nausea or vomiting�high heart rate�headache and a fever �headache with a stiff neck or stiff back





EMPAVELI is a medicine that can affect your immune system and can lower the ability of your immune system to fight infections.�EMPAVELI may increase your chance of getting serious and life‑threatening meningococcal infections that may quickly become life‑threatening and cause death if not recognized and treated early.�EMPAVELI may also increase the risk of getting serious infections caused by certain bacteria such as Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B. Serious infections may quickly become life‑threatening and cause death if not recognized and treated early. – You must be vaccinated against these bacteria at least 2 weeks before your first dose of EMPAVELI if you have not already had these vaccines.


What is the most important information I should know about EMPAVELI™ (pegcetacoplan)?



16

What is the most important information I should know about EMPAVELI? (cont’d)

Before you take EMPAVELI, it's important to have all your prior vaccination records readily available before starting treatment with EMPAVELI�You must be vaccinated against these bacteria at least 2 weeks before your first dose of EMPAVELI if you have not already had these vaccines� If your healthcare provider decides that urgent treatment with EMPAVELI is needed, you should receive the required vaccinations as soon as possible

� If you have not been vaccinated and EMPAVELI therapy must be started immediately, you should receive 2 weeks of antibiotics with your vaccinations � If you have been vaccinated against these bacteria in the past, you might need additional vaccinations before starting EMPAVELI. Your healthcare provider will decide if you need additional vaccinations�Vaccines reduce the risk of serious infections, but do not prevent all serious infections

Call your healthcare professional or seek emergency medical care right away if you have any new signs or symptoms of a serious infection:

�fever with or without shivers or the chills �fever and a rash�shortness of breath�extreme pain or discomfort

�headache with nausea or vomiting�high heart rate�headache and a fever �headache with a stiff neck or stiff back �confusion

�muscle aches with flu-like symptoms �clammy skin�eyes sensitive to light




17


EMPAVELI REMS program ensures your safety is a top priorityEMPAVELI 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 professional 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�A Patient Safety Card

– You’ll carry this card with you at all times during treatment and for 2 months after your last EMPAVELI dose. Your risk of serious infection may continue for several weeks after your last dose of EMPAVELI. It is important to show this card to any healthcare professional who treats you

�Appropriate vaccinations

Who should not take EMPAVELI? Do not take EMPAVELI if you:

�are allergic to pegcetacoplan or any of the ingredients in EMPAVELI�have not been vaccinated against Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B, unless your healthcare provider decides that urgent treatment with EMPAVELI is needed�have a serious Streptococcus pneumoniae, Neisseria meningitidis, or Haemophilus influenzae type B infection

Before you take EMPAVELI, tell your doctor about all 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 – females who are able to become pregnant should use an effective method of birth control (contraception) during treatment with EMPAVELI and for 40 days after the final dose




18

In the 16-week study, injection-site reactions and diarrhea were not severe and did not stop people from taking EMPAVELI.�These are not all the possible side effects of EMPAVELI. Be sure to tell your doctor 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

�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 final dose

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

Most common side effects

Abdominal pain

Tiredness

Injection-site reactions

Respiratory tract infection

Diarrheamy infusion studio

Infections


Before you take EMPAVELI, tell your doctor about all your medical conditions, including if you (cont’d):

Viral infection




http://www.fda.gov/medwatch

19

Your healthcare provider should show you how to prepare and infuse EMPAVELI before you use it for the first time. Use EMPAVELI exactly as your healthcare provider tells you.

* A subcutaneous infusion is given in the fatty tissue, just below the skin. †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.

for about 30 minutes (if using 2 infusion sites) or

60 minutes (if using 1)

2XA WEEK†

Transitioning to EMPAVELIFor the first 4 weeks, you will receive EMPAVELI and your current dose of eculizumab.

After 4 weeks, STOP eculizumab and only take EMPAVELI.

First

eculizumab + EMPAVELI

4 weeks

This process helps reduce the risk of hemolysis during your treatment transition.

If you are changing treatment from ravulizumab, you should start taking EMPAVELI no more than 4 weeks after your last dose of ravulizumab.

Take your treatment wherever is most convenient for youEMPAVELI is a 1080-mg subcutaneous self-infusion*

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 infection. Carry it with you at all times during treatment and for 2 months after your last EMPAVELI dose. It is important to show this card to any healthcare provider to help them diagnose and treat you quickly.Please see Important Safety Information, including Boxed WARNING regarding risk of serious infections, on pages 23-25, full Prescribing Information, and Medication Guide.



20

How should I take EMPAVELI™ (pegcetacoplan)?�See the Instructions for Use included with your medication�Before you begin treatment, a healthcare professional should show you how to prepare and infuse EMPAVELI�Your healthcare provider will tell you how much EMPAVELI to infuse and how often to infuse. Do not infuse more or less than your healthcare provider tells you to� If you miss a dose of EMPAVELI, take the missed dose as soon as possible. Take your next dose at your regularly scheduled time� 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

We have resources to help support you when

taking EMPAVELI

Visit EMPAVELI.com/support

Self-infusion video and training

Support communications

My Infusion Tracker™ app to help manage treatment

Take your treatment wherever is most convenient for you (cont’d)

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.�have not been vaccinated against Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B, unless your healthcare provider decides that urgent treatment with EMPAVELI is needed.�have a serious infection caused by the bacteria above.



https://pi.apellis.com/files/IFU_Empaveli.pdf

http://EMPAVELI.com/support


21

ApellisAssist is a program designed to help you along your treatment journey

Every person with PNH is unique and has their own needs when starting a new treatment. That is why ApellisAssist was created—to provide a comprehensive support system that’s always with you during your treatment journey.

This program provides services and product resources including insurance support, education, training, as well as financial assistance for eligible patients.

Once you are enrolled in ApellisAssist, you will have access to a dedicated Care Coordinator through our specialty pharmacy, PANTHERx Rare. Patients will also have access to an Apellis Care Educator, who will provide self-infusion training and disease education.

Always at your side

A dedicated coordinator who will work with you one-on-one

to help address your insurance, financial, and logistical

questions around EMPAVELI

A compassionate care partner with a nursing background who provides self-infusion

training, education, and ongoing EMPAVELI support

Care EducatorCare Coordinator

Call at

1-866-MY-APL-ASSIST (1-866-692-7527)FROM 8 AM-8 PM ET, MONDAY-FRIDAY

They can also connect you to a PANTHERx Rare pharmacist who

is on call 24 hours per day.




22

Helps integrate treatment into your life

HSH TVExpert advice from interior designer Genevieve Gorder on how to set up

your at-home treatment area

My Infusion Studio A case to simply stow away all of your non-refrigerated

treatment supplies

Refrigerator StorageA secure storage solution for

your medication cartons

HOME SUITE HOME has tools to help you stay organized, store your medication, and get comfortable in your own space

You will have access to these tools once you enroll in ApellisAssist, but they are not a requirement. We hope that you will find them to be a helpful addition to your treatment journey.




INDICATION AND IMPORTANT SAFETY INFORMATION

What is EMPAVELI?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 can affect your immune system and can lower the ability of your immune system to fight infections.

�EMPAVELI may increase your chance of getting serious and life‑threatening meningococcal infections that may quickly become life‑threatening and cause death if not recognized and treated early.�EMPAVELI may also increase the risk of getting serious infections caused by certain bacteria such as Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B. Serious infections may quickly become life‑threatening and cause death if not recognized and treated early.

1. You must be vaccinated against these bacteria at least 2 weeks before your first dose of EMPAVELI if you have not already had these vaccines.

2. If your healthcare provider decides that urgent treatment with EMPAVELI is needed, you should receive the required vaccinations as soon as possible.

3. If you have not been vaccinated and EMPAVELI therapy must be initiated immediately, you should also receive 2 weeks of antibiotics with your vaccinations.

4. If you have been vaccinated against these bacteria in the past, you might need additional vaccinations before starting EMPAVELI. Your healthcare provider will decide if you need additional vaccinations.

5. Vaccines reduce the risk of serious infections, but do not prevent all serious infections. 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 and a rash�shortness of breath�extreme pain or discomfort

�headache with nausea or vomiting�high heart rate�headache and a fever�headache with a stiff neck or stiff back


Please see full Prescribing Information, including Boxed WARNING regarding risk of serious infections,and Medication Guide for additional information.

23



IMPORTANT SAFETY INFORMATION (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. It is important to show this card to any healthcare provider to 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, give you a Patient Safety Card about your risk of serious infections, and make sure that you are vaccinated.

Who should NOT take EMPAVELI?Do not take EMPAVELI if you:�are allergic to pegcetacoplan or any of the ingredients in EMPAVELI.�have not been vaccinated against Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B, unless your healthcare provider decides that urgent treatment with EMPAVELI is needed.�have a serious infection caused by the bacteria above.

Before you take EMPAVELI, tell your healthcare provider about all 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 final 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 final dose.

2424




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.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�blood in your urine �pain in the stomach (abdomen)�shortness of breath �blood clots�trouble swallowing �erectile dysfunction (ED)

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 �swelling of your face, tongue, or throat�trouble breathing or shortness of breath �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, viral infection, and tiredness.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.


25


http://www.fda.gov/medwatch


Image is an actor portrayal.Quote from a real EMPAVELI patient.

EMPAVELI was studied vs eculizumab in a head‑to‑head Phase 3 clinical trial in 80 people living with PNH. Compared to the beginning of the study, people taking EMPAVELI had a 2.37 g/dL increase in hemoglobin levels compared to a 1.47 g/dL decrease with eculizumab at Week 16. EMPAVELI showed a superior 3.84 g/dL difference in Hb levels vs eculizumab (P<0.0001).

“My hemoglobin is higher with EMPAVELI. I think about what’s happening in PNH, and this is so important to me.”




27

Glossary of terms

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 PNH, as the body is making more new red blood cells to replace the ones being destroyed by the immune system.C3A protein in the blood that acts as a central point of the complement system, regulating its activation.C5Another protein in the blood that is part of the complement system.Clinical trialA type of research study that tests how well treatments work in people, as well as possible side effects of treatments.Complement systemThe 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.

Extravascular hemolysis (EVH)Destruction of red blood cells that occurs in the liver or spleen.

FACIT-Fatigue ScaleThis tool consists of a number of questions that are used to assign a score to measure a person’s experience with fatigue.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.HemolysisThe “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.




28

Immune systemA 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.

Open-label clinical trialA type of study in which both the healthcare providers and the patients are aware of the drug or treatment being given.

ProteinsMolecules 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 cellsA common type of blood cell. Their job is to carry oxygen, using an important molecule, hemoglobin, around the body.

ReticulocyteAn immature, or young, red blood cell.

U/LSome test results are reported in U/L, or units per liter. This is a measurement of how many units there are in a given liter.

X 109 cells/LA unit of measurement where the number of cells is multiplied by 109 power. This helps notate cell counts that are in the billions.

Glossary of terms (cont’d)





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