What to Expect from Treatment

How is Fabrazyme treatment given?

Fabrazyme is administered by intravenous (IV) infusion. This is a process that involves injecting the drug into a vein, directly into the bloodstream. Like other protein-based medications, Fabrazyme cannot be taken orally as a pill or liquid because proteins are destroyed in the digestive system. Infusion into the bloodstream bypasses your body’s digestive processes. Before the infusion begins, your healthcare provider may give you anti-fever medications (such as acetaminophen or ibuprofen) to help prevent reactions that your body may have to Fabrazyme. Infusion-associated reactions still occurred in some people even after receiving anti-fever medications, antihistamines, and oral steroids. Some reactions were serious.

What is the recommended dosage of Fabrazyme?

The amount of Fabrazyme you will be given is based upon how much you weigh. The recommended dosage of Fabrazyme is 1 milligram (mg) for each kilogram (kg) of body weight, given once every 2 weeks. For instance, a patient weighing 70 kg (about 154 pounds) would receive 70 mg of Fabrazyme every 2 weeks.

What should I expect during the infusion?

During the infusion, you can do things such as read a book, talk on the phone, write in your journal, talk with friends or family members who are with you, use the restroom – even take a nap if you wish. Your infusion time may be approximately 4 to 5 hours when you first start treatment. If you safely tolerate the infusion, your doctor may reduce your infusion time. Clinical studies have shown that over time, some patients can reduce their infusion times to between 2 and 3 hours. Infusion times vary among patients. Talk with your doctor about what to expect for your infusion time.

What should I tell my doctor before I start Fabrazyme?

Talk to your doctor about any of the situations below that apply to you, as they may affect how your doctor will choose to treat you.

Pregnancy or breast-feeding: If you think you are pregnant or if you plan to become pregnant, your doctor needs to know as soon as possible. Also, tell your doctor if you are breast-feeding.

Medical problems or allergies: Be sure your doctor is aware of any medical problems or allergies you have now or have had in the past, even if they are not related to Fabry disease.

Other medicines: Talk to your doctor about any medications you are taking, including over-the-counter medicines, vitamins and dietary supplements.

Can women be treated with Fabrazyme?

Clinical studies of Fabrazyme included twelve adult women and two girls (age 11). Although the safety and efficacy data available in female patients in clinical studies are limited, there is no indication that females respond differently to Fabrazyme than males.

The symptoms of Fabry disease vary more among females than they do in males. They may be mild or severe, and may affect some organ systems while sparing others. Serious complications may develop in some women, even if early signs and symptoms are mild. Talk with your doctor about whether treatment is right for you.

Fabrazyme has been shown to lower GL-3 in certain cells of the kidney, heart, and skin. However, research has not yet shown whether reducing the amount of GL-3 improves the signs and symptoms of Fabry disease.

Can children be treated with Fabrazyme?

If your child has Fabry disease, talk to his or her doctor about the right time to start treatment. The safety and effectiveness of Fabrazyme in patients ages 8 to 16 is similar to the safety and effectiveness seen in adults. Sixteen pediatric patients ages 8 to 16 were included in clinical studies of Fabrazyme. Patients younger than 8 years of age were not included in clinical studies so the safety and effectiveness of Fabrazyme in this age group has not been established.

Why are regular infusions important?

Since your body does not produce enough of the alpha-GAL enzyme on its own, it needs a regular supply of the replacement enzyme. That means Fabrazyme may be a long-term part of your treatment for Fabry disease. Regular infusions every 2 weeks help ensure that the body has an ongoing supply of enzyme to reduce the GL-3 in certain cells in the kidney, heart, and skin.

How can I explain the importance of infusions to my employer and others?

Infusions are likely to be regularly scheduled events in your life. Your infusions may need to occur during work hours. If your child receives Fabrazyme, his or her infusions may need to occur during school hours. You may decide to inform employers, teachers and others about your own or your child’s Fabry disease and the need for regular infusions. This is an important decision and you may want to talk with your healthcare provider about it. You can also call a Sanofi Genzyme Case Manager at 617-768-9000 (option 3), or toll-free 800-745-4447 (option 3) to request further information.

What happens if I miss an infusion?

If you miss an infusion, talk to your doctor about rescheduling your appointment. It may be necessary for you to plan ahead if you will be moving or traveling, so that you don’t miss an infusion. Talk with your doctor or Sanofi Genzyme Case Manager about the potential for arranging for treatment at your destination.

What should I expect from regular treatments with Fabrazyme?

Fabry disease affects people differently. Your doctor will probably want to see you regularly to check how you are responding to Fabrazyme treatment and to assess your overall health. It is important to remember that Fabrazyme is helping to clear build-up of GL-3. Keep in mind that GL-3 may have been building up in your cells for some time. However, research has not yet shown whether reducing the amount of GL-3 improves the signs and symptoms of Fabry disease.

What else can I do to take care of myself?

There are lifestyle changes you can make to maintain your health. For instance, you may want to minimize situations that cause you pain, such as physical exertion, sun or heat exposure, extreme temperature changes, or stress. You may also want to drink more liquids in hot weather or during physical activities. Avoid smoking, since it can increase your risk of breathing problems. If you have frequent stomach aches or gastrointestinal complaints, adjustments in your diet may help. Talk with your doctor about making these and other lifestyle changes.

Indication and Usage

Fabrazyme® (agalsidase beta) 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.

Important Safety Information

Fabrazyme can cause serious side effects, including:
Severe Allergic Reactions (anaphylaxis): Life-threatening severe allergic (anaphylactic) 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 or severe allergic reactions during their infusion.
  • These reactions have included: localized swelling of the face, mouth and throat, narrowing of breathing airways, low blood pressure, hives, difficulty swallowing, rash, trouble breathing, flushing, chest discomfort, itching and nasal congestion.
  • People who have experienced these reactions have required treatment including heart/lung resuscitation, oxygen, fluids given through the vein, hospitalization, and have needed treatment with inhaled drugs called beta-adrenergic agonists to help open the breathing airways, antihistamines, epinephrine (also known as adrenalin), and a medication given through the vein called a corticosteroid (or steroid) which helps to decrease the body’s allergic reaction by decreasing inflammation.
  • If you experience a severe allergic or anaphylactic reaction, your healthcare professional will immediately stop the infusion of Fabrazyme and provide you the necessary emergency medical treatment. Because of the possibility that severe allergic reactions may occur, appropriate medical support should be available during your Fabrazyme infusion.
Infusion-Associated Reactions: In clinical studies with Fabrazyme, 59% of patients experienced infusion-associated reactions during Fabrazyme administration, some of which were severe.
  • 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 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.

Pre-existing Heart Problems: 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.

Immune Response and Continued Treatment After Allergic Reaction: In the clinical studies, a few patients developed IgE antibodies or a reaction to an allergy skin test specific to Fabrazyme. IgE antibodies are usually produced by the body’s immune system during an allergic reaction. Your doctor should consider testing for IgE antibodies if you experience suspected allergic reactions. Providing Fabrazyme to patients who have experienced severe or serious allergic reactions to Fabrazyme should only be done after carefully considering the risks and benefits of continuing the treatment, and only under the direct supervision of a qualified healthcare professional and with appropriate medical support readily available.

Common and Other Possible Side Effects:
  • Common side effects reported in 20% or more of Fabrazyme treated patients in clinical studies compared to placebo were upper respiratory tract infection, headache, cough, burning and/or tingling sensation, fatigue, dizziness, swelling in the legs, and rash.
  • Serious and/or frequently occurring side effects (occurring in 5% or more of the patients) thought to be related to Fabrazyme in placebo-controlled and open-label clinical studies have included: chills, fever, feeling hot or cold, trouble breathing, nausea, flushing of the skin, headache, vomiting, burning and/or tingling sensation, fatigue, itching, pain in the hands and feet, high blood pressure, chest pain, throat tightness, abdominal pain, dizziness, rapid heart rate, nasal congestion, diarrhea, swelling in the legs, muscle pain, back pain, paleness of the skin, slow heart rate, hives, low blood pressure, face swelling, rash and sleepiness.
  • Other serious side effects that were seen in the clinical studies included stroke, pain, lack of muscle coordination, slow or irregular heartbeat, stopping of the heartbeat, decreased blood pumped by the heart, dizziness, and kidney problems resulting in too much protein leaving the body in the urine (nephrotic syndrome). These side effects also occur as part of Fabry disease.
  • Since Fabrazyme has been approved, there have been side effects that resulted in death that may or may not be related to the use of Fabrazyme. These included: the heart and/or lungs stop working (known as cardiorespiratory arrest, respiratory failure, and/or cardiac failure), life-threatening infection in the blood stream (known as sepsis), stroke, heart attack, kidney failure, and pneumonia. Some of these side effects were reported in Fabry disease patients with significant underlying disease.

The safety and effectiveness of Fabrazyme in patients younger than 8 years of age have not been studied.

Please see full prescribing information for Fabrazyme.