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Cindy and Amanda, Fabry Patients

FABRAZYME: TREATING FAMILIES AFFECTED BY FABRY DISEASE FOR OVER 15 YEARS

Fabrazyme is used to treat patients with Fabry disease. Fabrazyme lowers the amount of a substance called globotriaosylceramide (GL-3), which builds up in cells lining the blood vessels of the kidney and certain other cells.

The lowering of GL-3 suggests that Fabrazyme may improve how Fabry disease affects your body; however, a relationship of lower GL-3 to specific signs and symptoms of Fabry disease has not been proven.

Cindy and Amanda, Fabry Patients
 

How Fabrazyme Works

See how Fabrazyme works inside the body

Fabrazyme helps patients with Fabry disease by replacing a missing enzyme. 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 can help people with Fabry disease

In clinical studies, Fabrazyme cleared GL-3 buildup in certain cells of key organs (kidney, heart, skin).

Forty-four of the 58 patients completed an additional 54 months of treatment in an open label extension study, where everyone received Fabrazyme therapy.


• Fabrazyme cleared GL-3 buildup within 5 months of treatment. This means if you started treatment with Fabrazyme in early spring, by the end of summer your GL-3 levels could be lower

– After 5 months of treatment with Fabrazyme, most people achieved normal or the near-normal GL-3 levels in the cells lining the blood vessels of the kidney (69% of patients), heart (72%), and skin (100%)

» None of the patients on a placebo achieved clearance in the same cells in the kidneys. Only one placebo patient achieved clearance in the heart and one other placebo patient achieved clearance in the skin

» Patients received a score of 0 to 3 based on the amount of GL-3 in their cells. Most patients (69%) received a score of 0, meaning their GL-3 level was normal or near normal.

» The lowering of GL-3 suggests that Fabrazyme may improve how Fabry disease affects your body; however, a relationship of lower GL-3 to specific signs and symptoms of Fabry disease has not been proven.


Fabrazyme also clears GL-3 buildup in children 8 years and older*


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

• 14 boys with higher than normal GL-3 levels at the beginning of the study were treated with Fabrazyme

– 12 out of the 14 boys had GL-3 buildup in the cells lining the blood vessels of skin at the beginning of the study. At 24 and 48 weeks, GL-3 in the blood and in the skin were lowered to normal or near-normal levels

• Two girls who were treated with Fabrazyme had normal levels of GL-3 in their blood and certain cells in the skin at the start of the study, and GL-3 remained normal through 48 weeks


*Fabrazyme has not been studied in patients under the age of 8.

Safety Profile

Fabrazyme has a well established safety profile

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


Serious Side Effects

  • Approximately 1% of patients who have received Fabrazyme either during a clinical study or after Fabrazyme was approved have experienced severe allergic reactions (anaphylaxis) during their infusion
  • In clinical studies, 59% of patients experienced infusion-associated reactions during Fabrazyme treatment, some of which were severe
  • Infusion-associated reactions tended to decline in frequency with continued use of Fabrazyme in clinical studies; however, they may still occur despite extended duration of treatment
  • 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 infusion. Infusion-associated reactions have happened in some patients even after taking these medications before their infusions
  • 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

The most common side effects of Fabrazyme

Common side effects which occurred in ≥ 20% of patients treated with Fabrazyme, compared to placebo are:

Side effect Fabrazyme (n=80) % Placebo (n=60) %
Upper respiratory tract infection 44 30
Chills 43 12
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

Tell your healthcare provider about any side effects you experience.

Infusion-associated reactions

59% of patients who took Fabrazyme in clinical studies had infusion-associated reactions, some of which were severe. These infusion-associated reactions tended to decline on frequency with continued use of Fabrazyme. However, infusion-associated reactions may still occur even if you have taken Fabrazyme for a long time.

Infusion-associated reactions can be addressed in two ways:

1. Right before the infusion

  • Most people in clinical studies were pretreated with acetaminophen. Anti-fever and antihistamine medications are recommended for people who have had reactions to their infusions, before they start their next infusion
  • Infusion-associated reactions occurred in some people after receiving pretreatment with antipyretics, antihistamines, and oral steroids

2. During the infusion

  • Symptoms may be improved by slowing the infusion rate, briefly stopping the infusion, and/or giving more anti-fever and antihistamine medications, and/or steroids
  • If severe infusion-associated reactions happen, your healthcare professional will immediately stop the infusion of Fabrazyme and provide you the necessary emergency medical treatment

STARTING FABRAZYME

Setting expectations for treatment with Fabrazyme

If you and your doctor have decided that it is time for you to start Fabrazyme, learn about what you can expect. Fabrazyme can be given in a variety of settings so talk to your doctor about which option is best for you.

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


The most common side effects reported with Fabrazyme are infusion-associated reactions. Some reactions were severe. Infusion-associated reactions occurred in 59% of patients but tended to decline in frequency with continued use of Fabrazyme during clinical studies. However, infusion-associated reactions may still occur despite extended duration of Fabrazyme treatment.


Some people may need to take anti-fever and antihistamine medications right before their infusion. Infusion-associated reactions have happened in some people even after taking these medications and steroids before their infusions.


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


If severe infusion-associated reactions happen, your doctor should consider stopping the infusion right away and provide medical care for your condition. Because severe infusion-associated reactions may happen, medical support and treatment should be readily available during your infusion.



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, having concerns about side effects—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.


*Graph shows % of patients not achieving normal or near normal levels.

Number of Patients with Measurable GL-3 Deposits
in Certain Cells of the Kidney, Heart, and Skin
Kidney Heart Skin
Pretreatment 57 of 58 52 of 58 55 of 58
6-12 months 2 of 49 8 of 40 2 of 53
54+ months 0 of 8 2 of 8 5 of 36

58 patients, ages 16-61, participated in a clinical study where they received either Fabrazyme or placebo every 2 weeks for 5 months.

  • Most patients treated with Fabrazyme achieved normal or near normal GL-3 levels in the cells lining the blood vessels of the kidney (69% of patients), heart (72%), and skin (100%). In patients on placebo, none achieved clearance in the same cells of the kidney and only one patient achieved clearance in the heart and skin, respectively.
  • 44 of the 58 patients completed an additional 54 months of treatment with Fabrazyme in an open label extension study; patients who were initially on placebo were switched to Fabrazyme.
  • The lowering of GL-3 suggests that Fabrazyme may improve how Fabry disease affects your body; however, a relationship of lower GL-3 to specific signs and symptoms of Fabry disease has not been proven.