Narrator VO:

What is chronic granulomatous disease?

Patient VO:

Hi my name is Muris.

I live in Illinois with my brother, dad and mom.

In my free time, I like to play outside, play football and basketball with my friends.

I was diagnosed with CGD when I was 2 months old.

When I was old enough to understand what CGD was I felt nervous and scared.

I had a lot of questions too, like how long would I be on the medication, and what should I stay away from…

...but over time I learned a lot about CGD and how to manage my condition.

You may also have questions like I did…

...so let’s take a closer look to see what happens inside the body when you have CGD.

Narrator VO:

Chronic Granulomatous Disease, or CGD, is a rare, genetic disease that affects the immune system.

The immune system is a network of cells, tissues, and organs that help the body fight infection.

Blood cells called white blood cells are an important part of the immune system that help attack foreign Invaders…

...like germs. Normally, when germs enter the body…

...the immune system activates white blood cells to rush to the site of infection and attack the germs.

There are several different types of white blood cells.

One type is called a phagocyte. Phagocytes work by ingesting the germs and trapping them inside.

The phagocyte then uses a special killing mechanism called an “oxidative burst” to destroy the germs.

A healthy immune system usually prevents infections from becoming serious.

But people with CGD are at a greater risk of serious and potentially life-threatening bacterial and fungal infections because their immune system can’t fight them off as well.

In people with CGD, the phagocytes can still trap the germs…

...but their killing mechanism doesn’t work properly, so they can’t completely destroy bacteria and fungi.

The immune system sends more... ...and more white blood cells to fight the infection. But the infection doesn’t clear, and can become serious and life threatening.

The white blood cells just pile up forming a hard lump called a granuloma.

Granulomas can affect many parts of the body including the skin, urinary tract, stomach, and lymph nodes, resulting in complications like stomach pain, diarrhea, and weight loss.

Persistent, recurring infections are often serious and even life threatening...

...and can lead to pneumonia, liver and skin abscesses, inflammation of the colon - or colitis - and bone infections - or osteomyelitis.

Patient VO:

It’s important to remember that CGD is a serious condition and should be managed by working with your doctor. Now watch the next video to learn about available treatment options.

Narrator VO: For more information, speak with your doctor and visit ACTIMMUNE.com.

IMPORTANT SAFETY INFORMATION

What is ACTIMMUNE® (Interferon gamma 1-b) used for?

ACTIMMUNE is part of a drug regimen used to treat Chronic Granulomatous Disease, or CGD. CGD is a genetic disorder, usually diagnosed in childhood, that affects some cells of the immune system and the body’s ability to fight infections effectively. CGD is often treated (though not cured) with antibiotics, antifungals, and ACTIMMUNE.

ACTIMMUNE is also used to slow the worsening of severe, malignant osteopetrosis (SMO). SMO is a genetic disorder that affects normal bone formation and is usually diagnosed in the first few months after birth.

When should I not take ACTIMMUNE?

Don’t use ACTIMMUNE if you are allergic to interferon-gamma, E coli-derived products, or any ingredients contained in the product.

What warnings should I know about ACTIMMUNE?

At high doses, ACTIMMUNE can cause (flu-like) symptoms, which may worsen some pre-existing heart conditions.

ACTIMMUNE may cause decreased mental status, walking disturbances, and dizziness, particularly at very high doses. These symptoms are usually reversible within a few days upon dose reduction or discontinuation of therapy.

Bone marrow function may be suppressed with ACTIMMUNE, and decreased production of cells important to the body may occur. This effect, which can be severe, is usually reversible when the drug is discontinued or the dose is reduced.

Taking ACTIMMUNE may cause reversible changes to your liver function, particularly in patients less than 1 year old. Your doctor should monitor your liver function every 3 months, and monthly in children under 1 year.

In rare cases, ACTIMMUNE can cause severe allergic reactions and/or rash. If you experience a serious reaction to ACTIMMUNE, discontinue it immediately and contact your doctor or seek medical help.

What should I tell my healthcare provider?

Be sure to tell your doctor about all the medications you are taking.

Tell your doctor if you:

• are pregnant or plan to become pregnant or plan to nurse
• have a cardiac condition such as irregular heartbeat, heart failure, or decreased blood flow to your heart
• have a history of seizures or other neurologic disorders
• have, or have had, reduced bone marrow function. Your doctor will monitor these cells with blood tests at the beginning of therapy and at 3-month intervals on ACTIMMUNE therapy

What are the side effects of ACTIMMUNE?

The most common side effects with ACTIMMUNE are “flu-like” symptoms such as fever, headache, chills, muscle pain, or fatigue, which may decrease in severity as treatment continues. Bedtime administration of ACTIMMUNE may help reduce some of these symptoms. Acetaminophen may be helpful in preventing fever and headache.

What other medications might interact with ACTIMMUNE?

Some drugs may interact with ACTIMMUNE to potentially increase the risk of damage to your heart or nervous system, such as certain chemotherapy drugs. Tell your doctor about all other medications you are taking.

Avoid taking ACTIMMUNE at the same time as a vaccination.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

The risk information provided here is not comprehensive. To learn more, talk about ACTIMMUNE with your healthcare provider or pharmacist. The FDA-approved product labeling can be found at http://www.ACTIMMUNE.com or 1-866-479-6742.

Live action: In the last episode we learned about what CGD is and what happens inside the body when you have the condition. Along with staying healthy and being cautious about your environment treatments like Actimmune are available to help manage CGD

Narrator VO: Treatments are available to help manage chronic granulomatous disease, including antifungals, antibiotics, and ACTIMMUNE® (Interferon gamma-1b). Let’s talk about how it works.

ACTIMMUNE is the only FDA-approved biologic therapy that modifies the immune system for people with CGD.

ACTIMMUNE helps activate and strengthen other parts of the immune system to fight infection.

Antibiotics help prevent and fight infection caused by bacteria.

Antifungal medicines can help prevent and fight infection caused by fungi.

So how does ACTIMMUNE work?

ACTIMMUNE contains a protein called interferon gamma, which is naturally made by the body to fight infections.

Interferon-gamma activates several parts of the immune system.

The first thing it does...is it super charges the white blood cells ...to break down germs.

They also activate the other immune cells, helping them to attack and destroy the infection.

The effects of interferon-gamma also make the germ or infected cell easier for the immune system to find and attack.

ACTIMMUNE is given with a small needle as an injection... into the fatty layer right under the skin three times a week. ACTIMMUNE is given as an injection instead of a pill because stomach acid would break down the medicine before it could get to where it needs to be.

Remember, ACTIMMUNE can lead to fewer serious infections, which means less time in the hospital and more time to do the things you enjoy.

Live Action: It’s important that I manage my CGD so I created a schedule that works for me. For my treatment, I take my pills twice a day every day and I take my Actimmune 3 times a week. I set a phone reminder so that I don’t forget. I also know that if I ever have any questions I have a support team, like my doctor and my family to help me. And remember, stay strong and take it one day at a time.

Narrator VO: For more information, speak with your doctor and visit ACTIMMUNE.com.

IMPORTANT SAFETY INFORMATION

What is ACTIMMUNE® (Interferon gamma 1-b) used for?

ACTIMMUNE is part of a drug regimen used to treat Chronic Granulomatous Disease, or CGD. CGD is a genetic disorder, usually diagnosed in childhood, that affects some cells of the immune system and the body’s ability to fight infections effectively. CGD is often treated (though not cured) with antibiotics, antifungals, and ACTIMMUNE.

ACTIMMUNE is also used to slow the worsening of severe, malignant osteopetrosis (SMO). SMO is a genetic disorder that affects normal bone formation and is usually diagnosed in the first few months after birth.

When should I not take ACTIMMUNE?

Don’t use ACTIMMUNE if you are allergic to interferon-gamma, E coli-derived products, or any ingredients contained in the product.

What warnings should I know about ACTIMMUNE?

At high doses, ACTIMMUNE can cause (flu-like) symptoms, which may worsen some pre-existing heart conditions.

ACTIMMUNE may cause decreased mental status, walking disturbances, and dizziness, particularly at very high doses. These symptoms are usually reversible within a few days upon dose reduction or discontinuation of therapy.

Bone marrow function may be suppressed with ACTIMMUNE, and decreased production of cells important to the body may occur. This effect, which can be severe, is usually reversible when the drug is discontinued or the dose is reduced.

Taking ACTIMMUNE may cause reversible changes to your liver function, particularly in patients less than 1 year old. Your doctor should monitor your liver function every 3 months, and monthly in children under 1 year.

In rare cases, ACTIMMUNE can cause severe allergic reactions and/or rash. If you experience a serious reaction to ACTIMMUNE, discontinue it immediately and contact your doctor or seek medical help.

What should I tell my healthcare provider?

Be sure to tell your doctor about all the medications you are taking.

Tell your doctor if you:

• are pregnant or plan to become pregnant or plan to nurse
• have a cardiac condition such as irregular heartbeat, heart failure, or decreased blood flow to your heart
• have a history of seizures or other neurologic disorders
• have, or have had, reduced bone marrow function. Your doctor will monitor these cells with blood tests at the beginning of therapy and at 3-month intervals on ACTIMMUNE therapy

What are the side effects of ACTIMMUNE?

The most common side effects with ACTIMMUNE are “flu-like” symptoms such as fever, headache, chills, muscle pain, or fatigue, which may decrease in severity as treatment continues. Bedtime administration of ACTIMMUNE may help reduce some of these symptoms. Acetaminophen may be helpful in preventing fever and headache.

What other medications might interact with ACTIMMUNE?

Some drugs may interact with ACTIMMUNE to potentially increase the risk of damage to your heart or nervous system, such as certain chemotherapy drugs. Tell your doctor about all other medications you are taking.

Avoid taking ACTIMMUNE at the same time as a vaccination.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

The risk information provided here is not comprehensive. To learn more, talk about ACTIMMUNE with your healthcare provider or pharmacist. The FDA-approved product labeling can be found at http://www.ACTIMMUNE.com or 1-866-479-6742.

Randy: Hi, I’m Randy, and I’m going to show you how I take ACTIMMUNE. And believe me, if I can get comfortable with self-injecting, you can, too.

Super: RANDY, Lives with CGD

Art Card:

GET READY

Randy: When you first start ACTIMMUNE, your doctor or nurse will show you how to inject. If you ever have questions, that’s who to call first. You should also read the Patient/Caregiver Information that came with your ACTIMMUNE. Don’t try to inject until you’ve been trained and told you’re good to go.

Think of this video as a refresher—a little reminder on how to give an injection. OK? Let’s get started.

The first thing you do is lay out all your supplies on a clean surface.

Super: LAY OUT YOUR SUPPLIES

Randy: I like to take the vial out of the fridge about 15 minutes beforehand, so it has time to get up to room temperature.

Super: 15 (clock icon)

Randy: Always look over the vial. If it’s past the expiration date or if it looks cloudy, don’t use it. And don’t ever shake the vial.

Super: EXAMINE THE VIAL

• CHECK THE EXPIRATION DATE
• CHECK FOR CLOUDINESS
• DO NOT SHAKE THE VIAL

In addition to your vial of ACTIMMUNE, you’ll need alcohol swabs, alcohol-soaked cotton balls and a syringe and needle. Also a Sharps Container, which you’ll need for disposing of the syringe and needle later.

Super: (icons for alcohol swabs, syringe/needle, sharps container)

Art Card: MEASURE

Randy: Always wash your hands with soap before measuring your dose of ACTIMMUNE.

Super: ALWAYS WASH YOUR HANDS

Randy: I take off the red cap from the vial, then I wipe the top with an alcohol swab.

Randy: I take the syringe out of its package. Leaving on the plastic needle guard, I draw air into the syringe by pulling back on the plunger. I pull it back just to the mark for the dose of ACTIMMUNE my doctor told me to take.

I take off the needle guard and set it aside for the moment.

Randy: Holding the vial upright, I carefully stick the needle in through the rubber top.

Randy: Gently, I push the plunger down all the way so the air goes into the vial.

Randy: I hold the vial upside down with one hand, and with the other hand, I slowly pull the plunger out to the mark for the dose of ACTIMMUNE my doctor told me to take, drawing the correct amount of medicine into the syringe.

Randy: I take the needle out and replace the needle guard.

Art Card: SELECT

Randy: So I’ve measured my dose and I’ll inject in just a moment, but first I’m going to select an injection site.

It’s important to rotate the site with every injection, so that the skin has time to heal. The usual injection sites are the thighs…

Randy: …the belly…

Randy: …and the upper arms. My doctor told me to look for a place with some fat in it, which minimizes discomfort. If you select the belly, make sure you inject at least 2 inches away from the belly button.

Super: USUAL INJECTION SITES

• THIGHS
• BELLY
• UPPER ARMS

(icon for 2 inches away from belly button)

Art Card: INJECT

Randy: First, I clean the injection site with an alcohol swab.

I remove the needle guard, then I hold the syringe, sort of like a pencil.

Randy: With my other hand, I squeeze the skin to be injected between my fingers.

Randy: I try to relax, then I put the needle in with a quick, firm motion. It should go in at about a 45-degree angle.

Super: (icon for 45 degree angle)

Randy: Now that the needle is in, I pull back on the plunger just a little to see if blood comes into the syringe. There’s no blood in the syringe, so I’m ready to inject. But if I had seen blood, I would have removed the needle from my skin and tried again at another location.

Randy: Withdraw the needle quickly. Then apply pressure for a few seconds with a cotton ball or gauze.

Art Card: CLEAN UP

Randy: I’ve given the injection, but I’m not done until I’ve disposed of everything the right way.

Super: Visit ACTIMMUNE.com/support to explore all the resources and tools available.

It’s important to replace the needle guard and toss the needle and syringe in your Sharps Container. And finally, you need to dispose of any unused ACTIMMUNE the way your doctor told you to. Now you’re done. Good job! Randy: Now to help me keep track, I like to record the time and the location of the injection on this injection site calendar.

Super:

Logo: Horizon Patient Services logo

SUPPORT TO HELP YOU START AND STAY ON ACTIMMUNE® (INTERFERON GAMMA-1B)

HAVE QUESTIONS?

If you are enrolled in Horizon By Your Side:

Call, text, or email your Clinical Nurse Educator (CNE)

If you are not enrolled in Horizon By Your Side:

Call the CGD Nurse Advocate Call Center at 1 (833) 3-4MYCGD

Art Card: SAFETY INFORMATION

Voice Over: The most common side effects with ACTIMMUNE are “flu-like” symptoms such as fever, headache, chills, muscle pain, or fatigue, which may decrease in severity as treatment continues. Bedtime administration of ACTIMMUNE may help with some of these symptoms. Acetaminophen may be helpful in preventing fever and headache.

Voice Over: You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

Super: www.fda.gov/medwatch - 1 (800) FDA-1088

Please see the Full Prescribing Information and Important Safety Information available on this website. Please read the Information for Patients/Caregivers and consult your healthcare professional before giving an injection of ACTIMMUNE.

Art Card: REVIEW

Randy: Even after your doctor has trained you, being nervous about injecting ACTIMMUNE is totally normal. But trust me, it really does get better with practice.

Randy: If you want to review any of the steps in this process, please click the chapter titles at right.

Randy: I hope this video helps you become more comfortable with injecting. And if you still have questions, call your doctor.

IMPORTANT SAFETY INFORMATION

What is ACTIMMUNE® (Interferon gamma 1-b) used for?

ACTIMMUNE is part of a drug regimen used to treat Chronic Granulomatous Disease, or CGD. CGD is a genetic disorder, usually diagnosed in childhood, that affects some cells of the immune system and the body’s ability to fight infections effectively. CGD is often treated (though not cured) with antibiotics, antifungals, and ACTIMMUNE.

ACTIMMUNE is also used to slow the worsening of severe, malignant osteopetrosis (SMO). SMO is a genetic disorder that affects normal bone formation and is usually diagnosed in the first few months after birth.

When should I not take ACTIMMUNE?

Don’t use ACTIMMUNE if you are allergic to interferon-gamma, E coli-derived products, or any ingredients contained in the product.

What warnings should I know about ACTIMMUNE?

At high doses, ACTIMMUNE can cause (flu-like) symptoms, which may worsen some pre-existing heart conditions.

ACTIMMUNE may cause decreased mental status, walking disturbances, and dizziness, particularly at very high doses. These symptoms are usually reversible within a few days upon dose reduction or discontinuation of therapy.

Bone marrow function may be suppressed with ACTIMMUNE, and decreased production of cells important to the body may occur. This effect, which can be severe, is usually reversible when the drug is discontinued or the dose is reduced.

Taking ACTIMMUNE may cause reversible changes to your liver function, particularly in patients less than 1 year old. Your doctor should monitor your liver function every 3 months, and monthly in children under 1 year.

In rare cases, ACTIMMUNE can cause severe allergic reactions and/or rash. If you experience a serious reaction to ACTIMMUNE, discontinue it immediately and contact your doctor or seek medical help.

What should I tell my healthcare provider?

Be sure to tell your doctor about all the medications you are taking.

Tell your doctor if you:

• are pregnant or plan to become pregnant or plan to nurse
• have a cardiac condition such as irregular heartbeat, heart failure, or decreased blood flow to your heart
• have a history of seizures or other neurologic disorders
• have, or have had, reduced bone marrow function. Your doctor will monitor these cells with blood tests at the beginning of therapy and at 3-month intervals on ACTIMMUNE therapy

What are the side effects of ACTIMMUNE?

The most common side effects with ACTIMMUNE are “flu-like” symptoms such as fever, headache, chills, muscle pain, or fatigue, which may decrease in severity as treatment continues. Bedtime administration of ACTIMMUNE may help reduce some of these symptoms. Acetaminophen may be helpful in preventing fever and headache.

What other medications might interact with ACTIMMUNE?

Some drugs may interact with ACTIMMUNE to potentially increase the risk of damage to your heart or nervous system, such as certain chemotherapy drugs. Tell your doctor about all other medications you are taking.

Avoid taking ACTIMMUNE at the same time as a vaccination.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

The risk information provided here is not comprehensive. To learn more, talk about ACTIMMUNE with your healthcare provider or pharmacist. The FDA-approved product labeling can be found at http://www.ACTIMMUNE.com or 1-866-479-6742.

Art Card:INJECTION TRAINING FOR CAREGIVERS

Please listen to the Important Safety Information at the end of this video.

Jeannie: I was extremely nervous giving my son the injection the first time. I was afraid I was going to hurt him; I don’t like needles myself and he had been through so much already. But after the nurse educatior showed us how to do it, and I completed that first injection and now it’s part of our routine.

Jeannie/ Thaddaeus: Hi, I’m Jeannie, and I’m Thaddaeus and I have been taking ACTIMMUNE for two years and if I can get comfortable with it, you can, too. 

Super: JEANNIE, Caregiver

THADDAEUS, Living with CGD

Jeannie: When you first start ACTIMMUNE, your doctor or nurse will show you how to inject. If you ever have questions, that’s who you should call first. You should also read the Patient/Caregiver Information that came with your ACTIMMUNE. Don’t try to inject until you’ve been trained and told you’re good to go.

Let’s get started

Art Card: GET READY

Jeannie: Before we can begin the injection, we need do a few things.

I like to take the vial out of the fridge about 15 minutes beforehand, so it has time to get up to room temperature.

Super: 15 (clock icon)

It’s important to always look the vial over. If it’s past the expiration date or if its content looks cloudy, don’t use it. And don’t ever shake the vial.

Super: EXAMINE THE VIAL

• CHECK THE EXPIRATION DATE
• CHECK FOR CLOUDINESS
• DO NOT SHAKE THE VIAL

In addition to your vial of ACTIMMUNE, you’ll need alcohol swabs or alcohol-soaked cotton balls, and a syringe and needle. Also a Sharps Container, which you’ll need for disposing of the syringe and needle later

Art Card: MEASURE

Jeannie: Always wash your hands with soap before measuring your dose of ACTIMMUNE.

Jeannie: Take the red cap off the vial, then wipe the top with your alcohol swab.

Jeannie: Next, take the syringe out of its package. Leaving on the plastic needle guard, draw air into the syringe by pulling back on the plunger. I pull it back to the mark for the dose of ACTIMMUNE our doctor told us to take.

Then take off the needle guard, and set it aside for the moment.

Jeannie: Holding the vial upright, carefully stick the needle in through the rubber top and push the plunger down all the way so the air goes into the vial.

Jeannie: Now hold the vial upside down with one hand, and with the other hand, slowly pull the plunger out to the mark for the dose of ACTIMMUNE your doctor told you to take, drawing the correct amount of medicine into the syringe

Jeannie: Finally, take the needle out and replace the needle guard.

Art Card: SELECT

Jeannie: Now that we’ve measured our dose, we have to select an injection site. It’s important to rotate the site with every injection so that the skin has time to recover.

Jeannie: The usual injection sites are the thighs,…

Jeannie: …the belly,…

Jeannie: …and the upper arms. Our doctor told us to look for a place that’s padded by some fat underneath the skin, which helps reduce any pain. If you select the belly, make sure you inject at least 2 inches away from the belly button.

Jeannie: You have to work together on what works and what doesn’t work. We only do injections in the thighs because he’s not comfortable with the belly or the upper arms.

Art Card: INJECT

Jeannie: Now we’re ready to inject ACTIMMUNE. First, I sterilize the injection site with an alcohol swab. Then I remove the needle guard as I hold the syringe just like a pencil.

Jeannie: With my other hand, I gently squeeze the skin to be injected between my fingers to form a soft fold. I tell Thaddaeus to relax as much as he can…

Jeannie: …and then I push the needle in with a quick, firm motion at about a 45-degree angle.

Super: 45 (45 degree angle icon)

Jeannie: Now that the needle is in, I pull back on the plunger just a little to see if blood comes into the syringe. There’s no blood in the syringe, so I’m ready to inject. But if I had seen blood, I would have removed the needle from his skin and tried again at another location.

Jeannie: Slowly inject the ACTIMMUNE by gently pushing the plunger until the syringe is empty. This should take just a few seconds.

Withdraw the needle quickly. Then apply pressure for a few seconds with a cotton ball or gauze. Then put a bandage on, and that’s it.

Thaddaeus: Now that my injection is done and I’m back in action, we just need to dispose of everything properly.

Art Card: CLEAN UP

Jeannie: It’s important to replace the needle guard and toss the needle and syringe in your Sharps Container.

And finally, dispose of any unused ACTIMMUNE the way your doctor told you to. Now we’re done.

Art Card: REVIEW

Super: Visit ACTIMMUNE.com/support to explore all the resources and tools available.

Jeannie/Thaddaeus: Even after your doctor has taken you through the steps, being nervous about injecting is totally normal.

But trust me, it gets easier with practice.

At first I was scared of taking my ACTIMMUNE but now I’ve been practicing and it’s easier.

Jeannie: I’m extremely proud of the way Thaddaeus handles this. We’ve come a long way, he’s starting to learn how to do each step. He’s got to own this part of managing his disease.

Jeannie: If you want to review any of these steps again, please refer to the chapters listed on the screen.

Jeannie: I hope this video helps you become more comfortable.  And if you still have questions, call your doctor.

By the way, the Horizon Support Network will give you a Sharps Container at no cost. Call the number on the screen to reach a program coordinator.

Art Card:

Logo: Horizon Patient Services

HAVE QUESTIONS?

If your child is enrolled in Horizon By Your side: Call, text, or email your Clinical Nurse Educator (CNE)

Art Card:

HAVE QUESTIONS?

If your child is not enrolled in Horizon By Your Side: Call the CGD Nurse Advocate Call Center at 1 (833) 3-4MYCGD

Art Card: SAFETY INFORMATION

Voice Over: VO: The most common side effects with ACTIMMUNE are “flu-like” symptoms such as fever, headache, chills, muscle pain, or fatigue, which may decrease in severity as treatment continues. Bedtime administration of ACTIMMUNE may help with some of these symptoms. Acetaminophen may be helpful in preventing fever and headache.

Voice Over: You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

Super: www.fda.gov/medwatch - 1 (800) FDA-1088

Please see the Full Prescribing Information and Important Safety Information available on this website. Please read the Information for Patients/Caregivers and consult your healthcare professional before giving an injection of ACTIMMUNE.

Occasionally, a problem may develop at an injection site. Call your doctor or nurse if you see a lump, swelling, or bruising that doesn’t go away, or any signs of infection, such as pus, hot skin, or pain that does not go away.

IMPORTANT SAFETY INFORMATION

What is ACTIMMUNE® (Interferon gamma 1-b) used for?

ACTIMMUNE is part of a drug regimen used to treat Chronic Granulomatous Disease, or CGD. CGD is a genetic disorder, usually diagnosed in childhood, that affects some cells of the immune system and the body’s ability to fight infections effectively. CGD is often treated (though not cured) with antibiotics, antifungals, and ACTIMMUNE.

ACTIMMUNE is also used to slow the worsening of severe, malignant osteopetrosis (SMO). SMO is a genetic disorder that affects normal bone formation and is usually diagnosed in the first few months after birth.

When should I not take ACTIMMUNE?

Don’t use ACTIMMUNE if you are allergic to interferon-gamma, E coli-derived products, or any ingredients contained in the product.

What warnings should I know about ACTIMMUNE?

At high doses, ACTIMMUNE can cause (flu-like) symptoms, which may worsen some pre-existing heart conditions.

ACTIMMUNE may cause decreased mental status, walking disturbances, and dizziness, particularly at very high doses. These symptoms are usually reversible within a few days upon dose reduction or discontinuation of therapy.

Bone marrow function may be suppressed with ACTIMMUNE, and decreased production of cells important to the body may occur. This effect, which can be severe, is usually reversible when the drug is discontinued or the dose is reduced.

Taking ACTIMMUNE may cause reversible changes to your liver function, particularly in patients less than 1 year old. Your doctor should monitor your liver function every 3 months, and monthly in children under 1 year.

In rare cases, ACTIMMUNE can cause severe allergic reactions and/or rash. If you experience a serious reaction to ACTIMMUNE, discontinue it immediately and contact your doctor or seek medical help.

What should I tell my healthcare provider?

Be sure to tell your doctor about all the medications you are taking.

Tell your doctor if you:

• are pregnant or plan to become pregnant or plan to nurse
• have a cardiac condition such as irregular heartbeat, heart failure, or decreased blood flow to your heart
• have a history of seizures or other neurologic disorders
• have, or have had, reduced bone marrow function. Your doctor will monitor these cells with blood tests at the beginning of therapy and at 3-month intervals on ACTIMMUNE therapy

What are the side effects of ACTIMMUNE?

The most common side effects with ACTIMMUNE are “flu-like” symptoms such as fever, headache, chills, muscle pain, or fatigue, which may decrease in severity as treatment continues. Bedtime administration of ACTIMMUNE may help reduce some of these symptoms. Acetaminophen may be helpful in preventing fever and headache.

What other medications might interact with ACTIMMUNE?

Some drugs may interact with ACTIMMUNE to potentially increase the risk of damage to your heart or nervous system, such as certain chemotherapy drugs. Tell your doctor about all other medications you are taking.

Avoid taking ACTIMMUNE at the same time as a vaccination.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

The risk information provided here is not comprehensive. To learn more, talk about ACTIMMUNE with your healthcare provider or pharmacist. The FDA-approved product labeling can be found at http://www.ACTIMMUNE.com or 1-866-479-6742.

Irma, CNE: My name is Irma. I’m the clinical nurse educator.

When I go to the patient’s house, the first thing I do is I introduce myself and my role and why I’m there for that day. I give ‘em an opportunity to just share everything that they’ve been through.

Jeannie: I’m Jeannie, the mother of Thaddeus. My son is 9 years old, and he was diagnosed with CGD at 6 years old.

The great thing about the support services is that it helps you not to feel alone. Just having that person that you know that you can call, and they can come and visit, and they can get to know your child and know you.

Irma We offer education, support, and advocacy. They can pick up the phone and call us. We’re there with them.

Jeannie: One of the most important things is when she described the CGD to Thaddaeus to help him understand what was wrong with his system, what caused it. That it wasn’t his fault.

Irma: I let them know that it’s an inherited primary immunodeficiency disease. It affects the child’s ability to fight infection. It doesn’t mean that they have to be isolated from the world. It doesn’t affect who they are. They just need to be careful.

Jeannie: I encourage any new families to take advantage of the support services, ‘cause the support team is fantastic.

Ramone: I had a landscaping business going. I would bike to the different places. I was in, in great shape. And then all of a sudden, it was early September. I wasn’t feeling good. And I never really got sick, where I was staying in the house. I was always out. Always active. And now, I was in bed and it was, probably now was on my second week of being in bed.

And then it got so bad, that I couldn’t, I couldn’t breathe and so…I told my parents, I gotta get professional help. I can’t breathe.

So when they got me to the emergency room. They poured the oxygen on me. And that’s all I remember for about a month and a half to two months. I was in an induced coma, a medically-induced coma at the time.

And they didn’t know what it was. They just knew I was getting worse as time went on. And so, they, they told my parents that we don’t think he’s gonna make it.

Rachel: I don’t think I really understood that he might die. I just knew that everybody was crying a lot and that my brother…was in the ICU and he was intubated. And I just remember being scared.

Ramone: There was a convention of doctors dealing with infectious disease and so they brought my case up at one of the meetings and a doctor had asked if he could look at the slides…he did notice the CGD, and so that’s how they figured out what it was. And they were able to start treating it.

Ramone: I think the good thing is, is that my parents didn’t keep me in a box.

They took the precautions in terms of, you can’t be near things that are gonna make you sick, but they tried to get me into the routine that I had before I got sick.

Ramone: We certainly pushed the limits. And I know I really did.

Robin: So, it’s, it’s like, “Are you sure you should be doing that?” was a question that I phrased quite a bit.

Robin: Ramone didn’t let the CGD diagnosis define him. It wasn’t who he was. It’s something that he had.

Ramone: The lead doctor at the time, he took me aside and…he just said, “Ramone, what you’re doing is helping other little boys so that they don’t have to go through what you went through.”

VO: ACTIMMUNE works with antibiotics and antifungals as a Triple Therapy to help protect patients with CGD against serious infections.

Ramone: I take the…I take ACTIMMUNE…I try to be consistent with that.

To take the ACTIMMUNE, which is just the one shot, is a lot easier than what I went through some 30 years ago.

Ramone: I would highly recommend that you just put in some effort. Put in some work and learn about what you have and then learn about the resources that are available to help you.

I would also say just to remain positive.

Celebrate success when you see it because there are so many challenges in life and you go up, you go down. And so, if you can see an area where you can just be happy with what you achieve today, take a moment and celebrate that.

DORIS: We first noticed there was something wrong with Casey at the age of four months. Because he wasn’t doing well when he would eat. And drink his milk... He would regurgitate it and he would have a fever.

DORIS: I would continue to tell the doctors. That something is wrong with Casey.

RONALD: If it had not been for, ah, her voicing the concern for Casey’s condition, it could have easily gone, ah, un-, undetected.

DORIS: Once he was diagnosed with CGD…

I decided then, that from then on, I will not only look for resources …but start a journal, so that way I’ll be able to journal everything that’s happening with him.

CASEY: My parents …let me know …to be leery of bacteria and germs and make sure you take care of cuts and scrapes, because your body can’t fight off infections and, and bacteria like other kids can.

I couldn’t…play in the dirt or grass…When it came to playgrounds, if there were wood chips, I couldn’t play on that playground.

When it came to school, I would always carry hand sanitizer and wipes. And if there were kids coughing, I would either take my work home or I would wear a mask to school.

CASEY: When I kinda took over the reins of my own health, you know, when I would go see a doctor… I would always take that journal just in case there was something I didn’t necessarily know…I was able to flip through those pages, just to have a better way of talking about it.

PROTECTION AGAINST INFECTION

CASEY: My doctor prescribed ACTIMMUNE to my treatment regimen to help lower my risk of serious infection.

DORIS: To me ACTIMMUNE is like a protection for him.

DORIS: When Casey left for school and was on his own, that was one of the main things I told him that be careful and make sure you take your medication and be an advocate for your own self, because it only would take that one infection that can take your life.

CASEY: When it came to college…I had this newfound freedom. I didn’t have my parents calling and checking on me to …make sure I took my medicine….. I wasn’t taking it like I should have. And… I went and swam in a lake, when I knew I wasn’t supposed to, and, ah, I started getting real bad headaches…and I knew something was wrong.

When I got to the hospital, I don’t really remember anything but filling out the intake form, and then I passed out. Well, my parents rushed to the hospital and the neurosurgeon …he was like, hey, there’s nothing we can do... And my mom, she just fought and fought, and she was not going to leave that hospital until he did something…

And I ended up having, ah, what’s called a craniotomy and …now I live with a titanium mesh plate in my head.

DORIS: This journey has been really long and hard and trying. And it has taken us through 18 doctors, 42 surgeries and procedures…

RONALD:. I could not even imagine myself enduring the things that he’s gone through. I’m so very proud of him

DORIS: I cannot express how great, how great of a feeling he gives me just watching him, and I get teary-eyed just watching him …knowing what all he’s gone through.

DORIS: Son, you are my super hero.

Art Card: After what Casey calls his “aha moment” in college, he started taking his medicine regulary

He graduated from college and went on to earn his master’s degree

Today he shares his story with the CGD community, hoping others will learn from his journey

Art Card: Get support and resources
1(877) 305-7704

Art Card: Logo: ACTIMMUNE® (Interferon gamma-1b)

Super: Horizon sign-off

IMPORTANT SAFETY INFORMATION

What is ACTIMMUNE® (Interferon gamma 1-b) used for?

ACTIMMUNE is part of a drug regimen used to treat Chronic Granulomatous Disease, or CGD. CGD is a genetic disorder, usually diagnosed in childhood, that affects some cells of the immune system and the body’s ability to fight infections effectively. CGD is often treated (though not cured) with antibiotics, antifungals, and ACTIMMUNE.

ACTIMMUNE is also used to slow the worsening of severe, malignant osteopetrosis (SMO). SMO is a genetic disorder that affects normal bone formation and is usually diagnosed in the first few months after birth.

When should I not take ACTIMMUNE?

Don’t use ACTIMMUNE if you are allergic to interferon-gamma, E coli-derived products, or any ingredients contained in the product.

What warnings should I know about ACTIMMUNE?

At high doses, ACTIMMUNE can cause (flu-like) symptoms, which may worsen some pre-existing heart conditions.

ACTIMMUNE may cause decreased mental status, walking disturbances, and dizziness, particularly at very high doses. These symptoms are usually reversible within a few days upon dose reduction or discontinuation of therapy.

Bone marrow function may be suppressed with ACTIMMUNE, and decreased production of cells important to the body may occur. This effect, which can be severe, is usually reversible when the drug is discontinued or the dose is reduced.

Taking ACTIMMUNE may cause reversible changes to your liver function, particularly in patients less than 1 year old. Your doctor should monitor your liver function every 3 months, and monthly in children under 1 year.

In rare cases, ACTIMMUNE can cause severe allergic reactions and/or rash. If you experience a serious reaction to ACTIMMUNE, discontinue it immediately and contact your doctor or seek medical help.

What should I tell my healthcare provider?

Be sure to tell your doctor about all the medications you are taking.

Tell your doctor if you:

• are pregnant or plan to become pregnant or plan to nurse
• have a cardiac condition such as irregular heartbeat, heart failure, or decreased blood flow to your heart
• have a history of seizures or other neurologic disorders
• have, or have had, reduced bone marrow function. Your doctor will monitor these cells with blood tests at the beginning of therapy and at 3-month intervals on ACTIMMUNE therapy

What are the side effects of ACTIMMUNE?

The most common side effects with ACTIMMUNE are “flu-like” symptoms such as fever, headache, chills, muscle pain, or fatigue, which may decrease in severity as treatment continues. Bedtime administration of ACTIMMUNE may help reduce some of these symptoms. Acetaminophen may be helpful in preventing fever and headache.

What other medications might interact with ACTIMMUNE?

Some drugs may interact with ACTIMMUNE to potentially increase the risk of damage to your heart or nervous system, such as certain chemotherapy drugs. Tell your doctor about all other medications you are taking.

Avoid taking ACTIMMUNE at the same time as a vaccination.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

The risk information provided here is not comprehensive. To learn more, talk about ACTIMMUNE with your healthcare provider or pharmacist. The FDA-approved product labeling can be found at http://www.ACTIMMUNE.com or 1-866-479-6742.

Art Card: Hear about her life with CGD and treatment with ACTIMUNE® (Interferon gamma-1b)

Please listen to the Important Safety Information at the end of this video. Individual experiences may vary with ACTIMMUNE.

Caitlin: Lives with autosomal recessive CGD I was diagnosed at 7 and I was having a lot of belly pain and …my mom and my dad… took me to the hospital…and then they realized, oh, maybe it’s her appendix. So they …decided to do exploratory surgery and they found …a large granuloma on my intestines, so they …started to do more tests, digging deeper and deeper and about a month later, I was diagnosed.

When I was growing up, I didn’t really have any limits as to what I could do. So, I was able to do things like Girl Scouts and go camping… I just had to be cautious.

ART CARD: STAYING PROTECTED

To manage my CGD, my doctor prescribed ACTIMMUNE and antibacterial and antifungal medications to lower my risk of serious infections.

My parents were really good about teaching me … the importance of taking my medicine. We had these pill containers they set up for me, so I know when I was supposed to take what.

When I first started taking ACTIMMUNE when I was about 7 years old…

I was terrified...to get the shots …

My parents sometimes even had to hold me down. I didn’t like it. I mean, I still don’t like it today, but obviously…

it only takes one infection for my life to spiral out of control.

I’ve heard a lot of families talk about how their kids don’t wanna become independent...My parents passed the reins over to me at a young age and I think that really helped me.

I followed rules …that we were supposed to…I guess it’s the teacher in me… I never really had any slip-ups in college that were CGD-related with my health. So, I was really thankful for that.

I’m most proud of the fact that I’ve had a normal life. I’m no different than anybody else. You know, I went to high school, went to college, got a job.

I think about the positive and how well I’m doing ...and what I’ve accomplished rather than, oh, I’ve been in the hospital for this amount of time, or I missed this much school, or I’m weird because of this. So, staying focused on the positive… has been really, really beneficial for me.

Art Card: Get support and resources

1(877) 305-7704

Logo: ACTIMMUNE^® (Interferon gamma-1b)

Super: Horizon sign-off

Art Card: SAFETY INFORMATION

IMPORTANT SAFETY INFORMATION

What is ACTIMMUNE® (Interferon gamma 1-b) used for?

ACTIMMUNE is part of a drug regimen used to treat Chronic Granulomatous Disease, or CGD. CGD is a genetic disorder, usually diagnosed in childhood, that affects some cells of the immune system and the body’s ability to fight infections effectively. CGD is often treated (though not cured) with antibiotics, antifungals, and ACTIMMUNE.

ACTIMMUNE is also used to slow the worsening of severe, malignant osteopetrosis (SMO). SMO is a genetic disorder that affects normal bone formation and is usually diagnosed in the first few months after birth.

When should I not take ACTIMMUNE?

Don’t use ACTIMMUNE if you are allergic to interferon-gamma, E coli-derived products, or any ingredients contained in the product.

What warnings should I know about ACTIMMUNE?

At high doses, ACTIMMUNE can cause (flu-like) symptoms, which may worsen some pre-existing heart conditions.

ACTIMMUNE may cause decreased mental status, walking disturbances, and dizziness, particularly at very high doses. These symptoms are usually reversible within a few days upon dose reduction or discontinuation of therapy.

Bone marrow function may be suppressed with ACTIMMUNE, and decreased production of cells important to the body may occur. This effect, which can be severe, is usually reversible when the drug is discontinued or the dose is reduced.

Taking ACTIMMUNE may cause reversible changes to your liver function, particularly in patients less than 1 year old. Your doctor should monitor your liver function every 3 months, and monthly in children under 1 year.

In rare cases, ACTIMMUNE can cause severe allergic reactions and/or rash. If you experience a serious reaction to ACTIMMUNE, discontinue it immediately and contact your doctor or seek medical help.

What should I tell my healthcare provider?

Be sure to tell your doctor about all the medications you are taking.

Tell your doctor if you:

• are pregnant or plan to become pregnant or plan to nurse
• have a cardiac condition such as irregular heartbeat, heart failure, or decreased blood flow to your heart
• have a history of seizures or other neurologic disorders
• have, or have had, reduced bone marrow function. Your doctor will monitor these cells with blood tests at the beginning of therapy and at 3-month intervals on ACTIMMUNE therapy

What are the side effects of ACTIMMUNE?

The most common side effects with ACTIMMUNE are “flu-like” symptoms such as fever, headache, chills, muscle pain, or fatigue, which may decrease in severity as treatment continues. Bedtime administration of ACTIMMUNE may help reduce some of these symptoms. Acetaminophen may be helpful in preventing fever and headache.

What other medications might interact with ACTIMMUNE?

Some drugs may interact with ACTIMMUNE to potentially increase the risk of damage to your heart or nervous system, such as certain chemotherapy drugs. Tell your doctor about all other medications you are taking.

Avoid taking ACTIMMUNE at the same time as a vaccination.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

The risk information provided here is not comprehensive. To learn more, talk about ACTIMMUNE with your healthcare provider or pharmacist. The FDA-approved product labeling can be found at http://www.ACTIMMUNE.com or 1-866-479-6742.

GROWING UP WITH CGD

EVAN:
Since I was young, my parents have been always been trying to tell me and remind me about CGD.
They have been extremely vigilant, very active with helping me and caring for me.

KEITH:
We started giving Evan the ACTIMMUNE injections when he was very young, around 2 years old.
But I think the most important thing is just to listen to your child.
And Evan would tell us, you know, when he we started, he did not want to watch.
He wanted a countdown.
We did it and we were done.
When he got a little older, he had to watch it.
He couldn't do it unless he was watching and until he was ready and he would tell us, OK, it's time.

EVAN:
When I was younger, my dad would tell me to help me understand that I always love to build and create.
So he would tell me the instructions for these building sets.
He would tell me that in my body, in my instructions, I'm missing 1 certain step. And the medicines that I'm taking add in that step to complete the set.

THERESA:
I pictured Evan eventually giving him the shot himself.
Knew he could do it.
Thought it was going to take many, many steps and and that's OK.
But eventually he would get there.
I was really excited for him to go ahead and and start.
You can do it.
I know you can.

EVAN
As soon as I was into my teenage years, I was like, OK, well, why do I have to take this medicine?
Like I'm not sick?
And all these doctors were telling me, oh, you're doing so good.
Keep it up.
I didn't realize that these medicines were the ones that were actually keeping me healthy.
And once my dad started transferring over to me, telling me, OK, now you have to remember.
You have to do your medicine or you have to come get me to help you with the shot. He told me I I am need to be on top of this. But I would.
I'd be like, oh, I could I could miss one.
I can miss.
I can miss some.
I could Oh, I just forgot.
That's OK.
I forgot.
And then that led to me probably like 2019 or 2020.
I don't remember when exactly, but I got so sick.
It was building inside of me, this abscess in my liver.
But it created just a whole, like, whole bunch of gunk in there because I wasn't taking my medicine like I should be.
And it actually sent me to the hospital for around like two or three weeks.
And from there, the whole recovery process was months and months.
And only after that did I realize, like, OK, maybe my parents were right.
Maybe, maybe this medicine stuff is really, I need this.
Like this is important.

OUR EXPERIENCE WITH ACTIMMUNE

THERESA
It all starts with learning at a young age instead of just, you know, always having the parent do it, introducing, you know what it is, how to do it, why you're taking it. But talking about all the aspects of it, the process, the ordering, what goes into it, I think those are all important pieces to eventually administering it yourself.

KEITH
When he was ready to do it on his own, we we knew, you know, he's going off to college, he's going to have to figure this out.
And so he started with getting the injection ready with the cap on the needle.
He touched his leg and he did that, you know, once or twice and then the next time with the cap off, he touched the needle to his leg.
And that's, you know, that's not easy to do.

EVAN
He would take out fruit that was old or expired and he would have me fill up the vial of the injection with water and then just give the fruit and have the action of giving a shot to something until I was ready for actually taking the ACTIMMUNE and giving it to myself.

KEITH
I'm exaggerating, but I think it took 1/2 an hour.
When he was done, he looked up at me and he said Dad, you're fired.
And he's been doing his injections ever since.

ADVICE FOR CAREGIVERS

KEITH
Letting go is very hard for parents for well, it was hard for me.
But it's it's necessary and it it takes years. Advice I would have for parents about when it's the right time for your child to take over their responsibility for their medication.
They're going to do it at some point. It's it's not a either or.
So it's a matter of finding the time when that's right for you and for your child of when you need to stop and let them have responsibility and responsibility.
They will take great pride in that.
And I think Evan realized he took great ownership when it was his responsibility.
He wasn't doing it for us anymore.
He was doing it for himself.

EVAN
For any parents I would just say as long as your kid is showing that they could, they could be responsible for it.
I think that you should trust them because once my parents showed me that they could trust me to do my medicine,
especially my shots and my pills, it got so much easier to do everything, 'cause I didn't feel like I was being forced.
I was in the position where I understood and I knew that this was under my control, approved uses and important safety information.

IMPORTANT SAFETY INFORMATION

What is ACTIMMUNE® (Interferon gamma 1-b) used for?

ACTIMMUNE is part of a drug regimen used to treat Chronic Granulomatous Disease, or CGD. CGD is a genetic disorder, usually diagnosed in childhood, that affects some cells of the immune system and the body’s ability to fight infections effectively. CGD is often treated (though not cured) with antibiotics, antifungals, and ACTIMMUNE.

ACTIMMUNE is also used to slow the worsening of severe, malignant osteopetrosis (SMO). SMO is a genetic disorder that affects normal bone formation and is usually diagnosed in the first few months after birth.

When should I not take ACTIMMUNE?

Don’t use ACTIMMUNE if you are allergic to interferon-gamma, E coli-derived products, or any ingredients contained in the product.

What warnings should I know about ACTIMMUNE?

At high doses, ACTIMMUNE can cause (flu-like) symptoms, which may worsen some pre-existing heart conditions.

ACTIMMUNE may cause decreased mental status, walking disturbances, and dizziness, particularly at very high doses. These symptoms are usually reversible within a few days upon dose reduction or discontinuation of therapy.

Bone marrow function may be suppressed with ACTIMMUNE, and decreased production of cells important to the body may occur. This effect, which can be severe, is usually reversible when the drug is discontinued or the dose is reduced.

Taking ACTIMMUNE may cause reversible changes to your liver function, particularly in patients less than 1 year old. Your doctor should monitor your liver function every 3 months, and monthly in children under 1 year.

In rare cases, ACTIMMUNE can cause severe allergic reactions and/or rash. If you experience a serious reaction to ACTIMMUNE, discontinue it immediately and contact your doctor or seek medical help.

What should I tell my healthcare provider?

Be sure to tell your doctor about all the medications you are taking.

Tell your doctor if you:

• are pregnant or plan to become pregnant or plan to nurse
• have a cardiac condition such as irregular heartbeat, heart failure, or decreased blood flow to your heart
• have a history of seizures or other neurologic disorders
• have, or have had, reduced bone marrow function. Your doctor will monitor these cells with blood tests at the beginning of therapy and at 3-month intervals on ACTIMMUNE therapy

What are the side effects of ACTIMMUNE?

The most common side effects with ACTIMMUNE are “flu-like” symptoms such as fever, headache, chills, muscle pain, or fatigue, which may decrease in severity as treatment continues. Bedtime administration of ACTIMMUNE may help reduce some of these symptoms. Acetaminophen may be helpful in preventing fever and headache.

What other medications might interact with ACTIMMUNE?

Some drugs may interact with ACTIMMUNE to potentially increase the risk of damage to your heart or nervous system, such as certain chemotherapy drugs. Tell your doctor about all other medications you are taking.

Avoid taking ACTIMMUNE at the same time as a vaccination.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

The risk information provided here is not comprehensive. To learn more, talk about ACTIMMUNE with your healthcare provider or pharmacist. The FDA-approved product labeling can be found at http://www.ACTIMMUNE.com or 1-866-479-6742.