How effective and safe is ACTIMMUNE®
in treating patients with
Chronic Granulomatous Disease (CGD)?

The International Chronic Granulomatous Disease Cooperative Study Group (ICGDCSG) conducted a controlled clinical trial in 128 patients (ages ranging from 1 to 44 years old) at 13 medical centers across 4 countries. The purpose of this clinical trial was to evaluate the safety and efficacy of ACTIMMUNE® in reducing the frequency and severity of serious infections in patients with CGD.1,2

Patients enrolled in the trial were randomly chosen to receive either ACTIMMUNE® or placebo, or “fake pill.” At least 85% of patients received prophylactic antibiotics in addition to ACTIMMUNE® or placebo. The number and timing of serious infections were tracked in all patients for up to 1 year. ACTIMMUNE® was administered 3 times weekly using the same dosing regimen that is recommended today. Average length of treatment was 8.9 months. The study was ended early when it was shown that ACTIMMUNE® significantly shortened time to serious infection (P=0.0036) compared to placebo. This was the primary goal of the trial.1,2 The results of the trial were published in the New England Journal of Medicine.1

65 patients were given placebo, and 63 patients were given ACTIMMUNE®. The study showed that patients taking ACTIMMUNE® had:

  • 67% reduction in relative risk of serious infections.1,2 That means the risk was lower compared to people taking placebo
    • It also means that patients taking ACTIMMUNE® may be able to spend more time doing things they enjoy
    67% reduction
  • 67% fewer inpatient hospital days*1,2
    • Fewer inpatient hospital days may mean more time at home with family and friends
    67% Fewer
  • 64% reduction in the total number and rate of serious infections.*1,2 This means they had fewer serious infections and there was more time in between each infection
  • 64% reduction

As shown in the clinical trial, a treatment benefit included a 53% reduction in the number of CGD patients with at least one serious infection* (ACTIMMUNE®: 14/63 vs. placebo: 30/65; P=0.002).

The beneficial effect of ACTIMMUNE® was demonstrated throughout a 12 month study, in which:

  • 77% of patients with CGD receiving ACTIMMUNE® were free of serious infection* compared to 30% of patients who received placebo (P=0.0006)

The average duration of therapy for these patients was 8.9 months.

Investigators concluded that ACTIMMUNE® is an effective and safe therapy for patients with Chronic Granulomatous Disease (CGD), since the therapy statistically reduced the frequency of serious infections.1

Patients receiving ACTIMMUNE® (interferon gamma-1b) versus placebo experienced a 67% reduction in relative risk of serious infections1,2

Reduction in serious infections

In a clinical trial, patients with CGD who received ACTIMMUNE® vs placebo required 67% fewer inpatient hospitalization days (ACTIMMUNE®: 497 days vs placebo: 1,493 days; P=0.02).1,2

Reduction in serious infections

In a clinical trial, patients with CGD who received ACTIMMUNE® vs placebo demonstrated a 64% reduction in total number and rate of serious infections,* including recurrent infections* (ACTIMMUNE®: 20 vs placebo: 56; P<0.0001).*1,2

*Serious infection was defined as a clinical event requiring hospitalization and intravenous (IV) antibiotics.

reduction in the total number and rate of serious infections

Safety and side effects of ACTIMMUNE®

The safety of ACTIMMUNE® was also evaluated during the same clinical trial. The researchers kept a record of all side effects that were experienced by patients in both the ACTIMMUNE® and placebo study groups.1

The most common side effects seen with ACTIMMUNE® are “flu-like” symptoms such as fever, headache, chills, myalgia (muscle pain), or fatigue, which may reduce in severity as treatment continues. Administering ACTIMMUNE® at bedtime may also help minimize some of these symptoms. Acetaminophen may be helpful in preventing fever and headache.

ACTIMMUNE® can cause severe allergic reactions and/or rash; flu-like symptoms, which may worsen pre-existing heart conditions; reversible changes to the nervous system (such as decreased mental status, walking disturbances, and dizziness); reversible severe bone marrow toxicity; decreased production of important cells in the body; and reversible changes to liver function (particularly in patients less than 1 year old).

If you develop a serious reaction discontinue treatment immediately; call your healthcare provider or seek medical help. Tell your healthcare provider if you have any allergies to interferon-gamma or E coli-derived products; a cardiac condition (such as irregular heartbeat, heart failure, or decreased blood flow to your heart); history of seizures or other neurologic disorders; or reduced bone marrow function. If you are pregnant or plan to become pregnant or plan to nurse you should consult your healthcare provider.

For a full list of side effects with ACTIMMUNE®, please see Full Prescribing Information.

Common adverse events in patients with CGD2

Adverse Events Box

This chart demonstrates side effects in patients with Chronic Granulomatous Disease taking ACTIMMUNE® injections or placebo, as directed during a clinical trial.1,2

Managing flu-like side effects2

  • Taking ACTIMMUNE® at bedtime may help reduce some of the ‘‘flu-like” symptoms
  • Acetaminophen may be used to prevent or reduce fever and headache
  • Side effects may decrease as treatment continues

If you have concerns regarding side effects or if severe reactions occur, call your healthcare provider immediately.

References: 1. The International Chronic Granulomatous Disease Cooperative Study Group. A controlled trial of interferon gamma to prevent infection in chronic granulomatous disease. N Engl J Med. 1991;324(8):509-516. 2. ACTIMMUNE® (Interferon gamma-1b) Full Prescribing Information. Roswell, GA: Horizon Pharma, Inc; 2015.

IMPORTANT SAFETY INFORMATION AND INDICATIONS

Important Information About ACTIMMUNE

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

ACTIMMUNE® (Interferon gamma 1-b) 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. You may also contact the Horizon Pharma Medical Information Department toll-free at 1-866-479-6742 or medicalinformation
@horizonpharma.com
.

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.