Fabrazyme

Name: Fabrazyme

Patient information

Patients should be informed that a Registry has been established in order to better understand the variability and progression of Fabry disease in the population as a whole and in women [see Use in Specific Populations], and to monitor and evaluate long term treatment effects of Fabrazyme (agalsidase beta) . The Registry will also monitor the effect of Fabrazyme (agalsidase beta) on pregnant women and their offspring. Patients should be encouraged to participate and advised that their participation is voluntary and may involve long-term follow-up. For more information visit www.fabryregistry.com or call (800) 745-4447.

Pregnancy & Lactation

Pregnancy Category: B

Lactation: Not known if distributed into breast milk, use caution

Pregnancy Categories

A:Generally acceptable. Controlled studies in pregnant women show no evidence of fetal risk.

B:May be acceptable. Either animal studies show no risk but human studies not available or animal studies showed minor risks and human studies done and showed no risk.

C:Use with caution if benefits outweigh risks. Animal studies show risk and human studies not available or neither animal nor human studies done.

D:Use in LIFE-THREATENING emergencies when no safer drug available. Positive evidence of human fetal risk.

X:Do not use in pregnancy. Risks involved outweigh potential benefits. Safer alternatives exist.

NA:Information not available.

Drug Interactions

Although certain medicines should not be used together at all, in other cases two different medicines may be used together even if an interaction might occur. In these cases, your doctor may want to change the dose, or other precautions may be necessary. Tell your healthcare professional if you are taking any other prescription or nonprescription (over-the-counter [OTC]) medicine.

Fabrazyme Interactions

No drug interactions have been studied by the manufacturer. However, you should tell your doctor about all the medicines you take including prescription and non-prescription medicines, vitamins, and herbal supplements. Not all drug interactions are known or reported and new drug interactions are continually being reported.

Fabrazyme Food Interactions

Medications can interact with certain foods. In some cases, this may be harmful and your doctor may advise you to avoid certain foods. In the case of Fabrazyme, there are no specific foods that you must exclude from your diet when receiving this medication.

Inform MD

Before taking Fabrazyme, tell your doctor about all of your medical conditions. Especially tell your doctor if you:

  • are allergic to Fabrazyme or to any of its ingredients
  • have heart problems
  • are pregnant or plan to become pregnant
  • are breastfeeding or plan to breastfeed

Tell your doctor about all the medicines you take including prescription and non-prescription medicines, vitamins, and herbal supplements.

What is agalsidase beta?

Agalsidase beta is a man-made form of the naturally-occurring alpha-galactosidase A enzyme. A deficiency of this enzyme is called Fabry disease. Agalsidase beta reduces deposits of globotriaosylceramide (GL-3) in the kidneys and certain other cells in the body.

Agalsidase beta is used in the treatment of Fabry disease.

Agalsidase beta may also be used for purposes other than those listed here.

How should I use agalsidase beta?

Agalsidase beta is injected into a vein through an IV. A healthcare provider will give you this injection.

Agalsidase beta is usually given once every 2 weeks. Follow your doctor's instructions.

While using agalsidase beta, you may need frequent medical tests.

What happens if I overdose?

Since this medication is given by a healthcare professional in a medical setting, an overdose is unlikely to occur.

Agalsidase beta side effects

Get emergency medical help if you have any of these signs of an allergic reaction:

  • skin rash, hives, flushing (warmth, redness, or tingly feeling);

  • trouble swallowing, chest discomfort, difficult breathing, feeling light-headed; or

  • swelling of your face, lips, tongue, or throat.

Many people have a severe reaction to the agalsidase beta infusion (side effects that occur during the injection). Tell your caregiver right away if you have any of these signs of an infusion reaction:

  • fever, headache, chills, stuffy nose, muscle pain, back pain, dizziness, drowsiness, tired feeling;

  • pale skin, feeling hot or cold, itching, numbness or tingly feeling, swelling in your hands or feet;

  • nausea, vomiting, tight feeling in your throat, stomach pain, diarrhea;

  • chest pain, fast or slow heart rate, feeling short of breath; or

  • a light-headed feeling, like you might pass out.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Advice to Patients

  • Importance of notifying clinician immediately if manifestations of infusion reaction (fever, rigors, chest tightness, hypertension, hypotension, pruritus, myalgia, dyspnea, urticaria, abdominal pain, headache) occur.1

  • Importance of informing patients that a registry has been established to monitor the variability and progression of Fabry’s disease and to evaluate long-term treatment effects of agalsidase beta.1

  • Importance of informing clinicians of existing or contemplated concomitant therapy, including prescription and OTC drugs, as well as any concomitant illnesses.a

  • Importance of women informing clinicians if they are or plan to become pregnant or plan to breast-feed.a

  • Importance of informing patients of other important precautionary information. (See Cautions.)

Commonly used brand name(s)

In the U.S.

  • Fabrazyme

Available Dosage Forms:

  • Powder for Solution

Therapeutic Class: Endocrine-Metabolic Agent

Pharmacologic Class: Enzyme

Fabrazyme Side Effects

Along with its needed effects, a medicine may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.

Check with your doctor immediately if any of the following side effects occur:

More common
  • Blurred vision
  • chest pain or discomfort
  • confusion
  • cough producing mucus
  • difficulty breathing
  • difficulty in moving
  • dizziness;
  • fatigue
  • faintness or lightheadedness when getting up from a lying or sitting position suddenly
  • feeling unusually cold shivering
  • headache
  • hives or welts
  • irregular heartbeat
  • itching
  • joint pain
  • muscle aching or cramping
  • muscle pains or stiffness
  • nervousness
  • pain
  • pounding in the ears
  • redness of skin
  • shortness of breath
  • skin rash
  • stomach pain
  • sweating
  • swelling of ankles, feet, and lower legs
  • swollen joints
  • tightness in chest
  • unusual tiredness or weakness
  • wheezing
Incidence unknown
  • Cloudy or bloody urine
  • decreased cardiac output
  • difficulty in speaking
  • double vision
  • feeling of constant movement of self or surroundings
  • high blood pressure
  • inability to move arms, legs, or facial muscles
  • inability to speak
  • no blood pressure or pulse
  • partial loss of hearing
  • pounding or rapid pulse
  • problems with muscle control or coordination
  • sensation of spinning
  • shakiness and unsteady walk
  • slow speech
  • stopping of heart
  • swelling of face
  • swelling of the lip or ear
  • throat tightness
  • trembling
  • unconsciousness

Some side effects may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects. Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:

More Common
  • Acid or sour stomach
  • belching
  • body aches or pain
  • body produces substance that can bind to drug making it less effective or cause side effects
  • burning, crawling, itching, numbness, prickling, "pins and needles" , or tingling feelings
  • congestion
  • discouragement
  • dryness or soreness of throat
  • fear
  • feeling sad or empty
  • fever, not related to infusion
  • heartburn
  • hoarseness
  • indigestion
  • irritability
  • lack of appetite
  • loss of interest or pleasure
  • nausea
  • pain or tenderness around eyes and cheekbones
  • paleness of skin
  • runny nose
  • sensation of change in temperature
  • skeletal pain
  • sneezing
  • stuffy nose; swelling of testes
  • tender, swollen glands in neck
  • trouble concentrating
  • trouble sleeping
  • trouble in swallowing
  • voice changes

Other side effects not listed may also occur in some patients. If you notice any other effects, check with your healthcare professional.

Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.

If OVERDOSE is suspected

If you think there has been an overdose, call your poison control center or get medical care right away. Be ready to tell or show what was taken, how much, and when it happened.

How do I store and/or throw out Fabrazyme?

  • If you need to store Fabrazyme at home, talk with your doctor, nurse, or pharmacist about how to store it.

Use in specific populations

Pregnancy

Pregnancy Category B -

There are no adequate and well-controlled studies of Fabrazyme use in pregnant women. Reproduction studies performed in rats at doses up to 30 times the human dose have revealed no evidence of impaired fertility or negative effects on embryo fetal development due to Fabrazyme.  Because animal reproduction studies are not always predictive of human response, this drug should be used during pregnancy only if clearly needed.

Women of childbearing potential should be encouraged to enroll in the Fabry patient registry. The registry will monitor the effect of Fabrazyme on pregnant women and their offspring.  For more information, visit  www.fabryregistry.com or call (800) 745-4447 [see Patient Counseling Information (17)].

Labor and Delivery

There is no information on the effect of Fabrazyme during labor and delivery. Pregnant females are encouraged to enroll in the Fabry registry [see Patient Counseling Information (17)].

Nursing Mothers

It is not known whether Fabrazyme is excreted in human milk. Because many drugs are excreted in human milk, caution should be exercised when Fabrazyme is administered to a nursing woman.

Nursing mothers should be encouraged to enroll in the Fabry registry [see Use in Specific Populations (8.1) and Patient Counseling Information (17)].

Pediatric Use

The safety and efficacy of Fabrazyme were assessed in a multi-national, multi-center, uncontrolled, open-label study in 16 pediatric patients with Fabry disease (14 males, 2 females), ages 8 to 16 years [see Clinical Studies (14)].  Patients younger than 8 years of age were not included in clinical studies.  The safety and efficacy in patients younger than 8 years of age have not been evaluated.

Geriatric Use

Clinical studies of Fabrazyme did not include sufficient numbers of subjects aged 65 and over to determine whether they respond differently from younger subjects.

Responses in Women

Fabry disease is an X-linked genetic disorder. However, some heterozygous women will develop signs and symptoms of Fabry disease due to the variability of the X-chromosome inactivation within cells.

A total of 12 adult female patients with Fabry disease were enrolled in two separate randomized, double-blind, placebo-controlled clinical studies with Fabrazyme, and two female pediatric patients with Fabry disease, ages 11 years, were evaluated in an open-label, uncontrolled pediatric study [see Use in Specific Populations (8.4) and Clinical Studies (14)].  Although the safety and efficacy data available in female patients in these clinical studies are limited, there is no indication that female patients respond differently to Fabrazyme compared to males.

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