Cindy and Amanda, Fabry Patients

ON YOUR FABRY DISEASE JOURNEY, COUNT ON FABRAZYME®.

FABRAZYME can be prescribed for people with Fabry disease, regardless of disease severity, enzyme activity, or type of genetic variation.

Fabrazyme is an enzyme replacement therapy (ERT) that supplies the enzyme that’s missing or deficient in Fabry disease. The fully functional enzyme helps clear GL-3 buildup in your cells.

Cindy and Amanda, Fabry Patients
 

How Fabrazyme Works

Fabrazyme helps to clear GL-3 buildup by replacing the missing enzyme in Fabry disease.

Watch this video to learn how Fabrazyme works inside the body.

Learn more about Fabry
disease and treatment
with Fabrazyme

fabrazyme patient brochure (english)
415kb

How Fabrazyme can Help

FABRAZYME has been proven to clear GL-3 buildup in clinical trials.

After 5 months of treatment with Fabrazyme in Study 1, most patients achieved GL-3 clearance resulting in trace, or nearly none, GL-3 inclusions in certain cells of their: Kidney: 69% (20/29) of Fabrazyme patients compared with 0% (0/29) of placebo patients; Heart: 72% (21/29) of Fabrazyme patients compared with 3% (1/29) of placebo patients; Skin: 100% (29/29) of Fabrazyme patients compared with 3% (1/29) of placebo patients.

Study 1 design: This study included 58 Fabry patients ages 16-61. Patients in this study received either Fabrazyme or placebo every 2 weeks for 5 months. Patients received a score of 0 to 3 based on the amount of GL-3 in their cells. Most people received a score of 0, meaning the GL-3 in their cells was nearly none or trace amounts.

Study 1 extension design: All 58 patients who completed Study 1 were treated with Fabrazyme every two weeks in an open-label extension study.

FABRAZYME was proven to clear GL-3 buildup in clinical trials.

Similar long-term GL-3 clearance was achieved at 4.5 years in the majority of patients taking Fabrazyme in this extension study.

In the extension study, patients had sustained GL-3 clearance in certain cells of the kidney (100%; 8/8) and heart (75%; 6/8) at 4.5 years.

A smaller percentage of individuals treated with Fabrazyme experienced a clinically significant event.*

A clinically significant event is defined as the first instance in any of the four categories below, which occurred after the study started.

A smaller percentage of people had heart, kidney, stroke events, or death.

A smaller percentage of people had heart, kidney, stroke events, or death with Fabrazyme

Study design: A randomized, double-blind, placebo-controlled, multinational, multicenter study of 82 patients (72 males and 10 females) with Fabry disease. Patients were 20 to 72 years of age with a median age of 45 years at baseline, a median age of 36 years at Fabry disease diagnosis, and at a median of 10 years at symptom onset.

*Renal, cardiac, or stroke events or death.

In children with Fabry disease aged 2-7 years, Fabrazyme normalized GL-3 in the blood.

An analysis of 24 children with elevated plasma GL-3 levels (i.e., > 7.03 ug/mL) taking Fabrazyme showed normalization of GL-3 levels in 91% (20/22), 95% (18/19), and 92% (12/13) of patients at 6, 12, and 24 months of treatment, respectively.

Study overview: In an observational study of 24 pediatric patients ages 2 to 7, normalization of plasma GL-3 was observed.

In children 8 years and older, Fabrazyme cleared GL-3.

Study overview: 16 pediatric patients with Fabry disease, aged 8-16 years, were evaluated in an open-label, uncontrolled study.

FABRAZYME cleared GL-3 buildup in children 8 years and older.

The most common adverse reactions (>20%) in patients aged 8-16 were headache, abdominal pain, sore throat, fever, nausea, vomiting, nasal inflammation, diarrhea, joint pain, and dizziness.

The rate of kidney function decline was studied in Fabrazyme-treated individuals.

1.7 - estimated difference in kidney function decline

estimated difference in the rate of
kidney function decline

The rate of renal function decline was assessed in Fabry disease patients aged ≥16 years. The mean slope of eGFR* was -1.5 mL/min/1.73m2/year in the Fabrazyme-treated group and -3.2 mL/min/1.73m2/year in the untreated group. The mean difference in eGFR between the two groups was 1.7 mL/min/1.73m2/year.

Study design: In a long-term observational study, the rate of decline in renal function (eGFR slope) was assessed in 122 patients with Fabry disease aged 16 years and older treated with Fabrazyme and matched to a historical cohort of untreated patients.

Safety Profile

The safety of FABRAZYME has been assessed in several studies.*

What are the most common side effects of FABRAZYME?

In clinical trials, common side effects that occurred in 20% or more of people treated with Fabrazyme and in more than 2.5% of people who received placebo, include:

Side effect Fabrazyme (n=80) % Placebo (n=60) %
Upper respiratory tract infection 53 42
Chills 49 13
Fever 39 22
Headache 39 28
Cough 33 25
Burning or tingling in hands and feet 31 18
Fatigue 24 17
Swelling in the limbs 21 7
Dizziness 21 8
Rash 20 10

n=number of patients.

*The safety of Fabrazyme has been assessed in 4 clinical studies involving 162 people with Fabry disease.

Talk to your doctor about any side effects you experience when taking Fabrazyme. Read below for more information about what to expect from your infusions.

Serious side effects

  • Life-threatening severe anaphylactic (allergic) or severe hypersensitivity reactions have been seen in patients during Fabrazyme infusions
  • Approximately 1% of patients who have received Fabrazyme either during a clinical study or after Fabrazyme was approved have experienced anaphylactic (allergic) or severe hypersensitivity reactions during their infusion
  • In clinical studies, 59% of patients experienced infusion-associated reactions during Fabrazyme treatment, some of which were severe
  • During the clinical trials, infusion-associated reactions occurred more frequently in patients who were positive for anti-Fabrazyme antibodies than in patients who did not have anti-Fabrazyme antibodies
  • People with advanced Fabry disease may have heart problems, which may put them at a higher risk for severe complications from infusion-associated reactions. These patients should be watched closely during their infusion if the decision is made to give them Fabrazyme

STARTING FABRAZYME

Treatment with FABRAZYME.

Fabrazyme can be given in a number of treatment settings, including:

Doctors office.

Doctor’s Office

Infusion Center.

Infusion Center

Home

Home

Talk to your doctor about which treatment setting is right for you.

Make your treatments a priority.

Make your treatments a priority

Fabrazyme is an infusion that's given to help prevent the buildup of GL-3. Fabrazyme keeps helping to clear GL-3 as long as you continue treatment.

Follow the Fabrazyme treatment plan prescribed by your doctor, even if you're not experiencing symptoms.

What to Expect From Treatment

Preparing for your infusion.

Your first infusion of Fabrazyme will be given in a doctor’s office or an infusion center. After several infusions, your doctor may give you the option of receiving Fabrazyme in your home.


To better incorporate infusions into your life, you may be able to request a specific day and/or time for your treatment. It is also a good idea to arrange for transportation to and from the infusion site—especially on your first visit.


Before you have your infusion, you may have an assessment by a healthcare professional, which can include asking you how you are currently feeling, measuring your weight, your heart rate, blood pressure, and temperature. Next, you may be given anti-fever (such as acetaminophen or ibuprofen) and antihistamine medications to help prevent or reduce infusion-associated reactions.


Following the assessment, the healthcare professional will start your IV, either through your port or through a vein in your arm. This is how Fabrazyme will get into your body during the infusion.


Managing infusion-related side effects.


Talk to your doctors about your potential for infusion-related side effects, and how they may be managed.

In clinical studies with Fabrazyme, 59% of patients experienced infusion-associated reactions during Fabrazyme administration, some of which were severe. Infusion-associated reactions are defined as adverse reactions occurring on the same day as your infusion. During the clinical trials, infusion-associated reactions occurred more frequently in patients who were positive for anti-Fabrazyme antibodies than in patients who did not have anti-Fabrazyme antibodies.


For patients who have had reactions to their infusions, it is recommended that they be given anti-fever and antihistamine medications right before their next infusions. Infusion-associated reactions have happened in some patients even after taking these medications before their infusions.


If an infusion-associated reaction occurs, slowing the infusion rate, stopping the infusion for a short time and/or giving more anti-fever and antihistamine medications and or steroids may improve the symptoms.


If severe infusion-associated reactions happen, your healthcare professional should consider stopping the Fabrazyme infusion right away and should provide medical care for your condition. Severe reactions are generally managed by giving antihistamine medications, corticosteroids, fluids through the vein, and/or oxygen when needed. Because severe infusion-associated reactions may happen, medical treatment should be readily available during your Fabrazyme infusion.


People with advanced Fabry disease may have heart problems which could put them at a higher risk for severe complications from infusion-associated reactions. These patients should be watched closely during their infusion if the decision is made to give them Fabrazyme.



Read about what to
expect before, during,
and after your infusion
with Fabrazyme

Your Infusion Experience
617KB

Staying on Treatment

Be sure to follow the treatment schedule your doctor recommends

Sometimes life’s challenges—being too busy, traveling away from home—can get in the way of receiving your Fabrazyme infusion. But it is important to follow the infusion schedule your doctor prescribes.

Fabrazyme keeps working when you keep taking it

Regular infusions of Fabrazyme every two weeks help ensure that the body has an ongoing supply of fully functional enzyme to clear GL-3 in certain cells of the kidney, heart, and skin.

Even if you don’t notice any symptoms, it’s important to continue to take Fabrazyme as directed, to help ensure that GL-3 doesn’t start to build up again.