Fabrazyme
Fabrazyme (agalsidase beta) is an enzyme replacement therapy (ERT) that's FDA-approved for the treatment of a rare, inherited condition called Fabry disease in adults and children. It's administered as an intravenous (IV) infusion by a healthcare provider every 2 weeks. The dosing is calculated by your weight. Side effects include a higher risk of infections, chills, fever, and headache.
What is Fabrazyme (agalsidase beta)?
What is Fabrazyme (agalsidase beta) used for?
Fabry disease in people 2 years of age and older
How Fabrazyme (agalsidase beta) works
Fabrazyme (agalsidase beta) is an enzyme replacement therapy for people with Fabry disease. It replaces the missing alpha-galactosidase A (alpha-Gal A) enzyme in the body. This enzyme helps clear the buildup of fats called globotriaosylceramide (GL-3) that causes damage to the body in Fabry disease.
Drug Facts
What are the side effects of Fabrazyme (agalsidase beta)?
Common Side Effects
- Upper respiratory tract infection (53%)
- Chills (49%)
- Fever (39%)
- Headache (39%)
- Cough (33%)
- Feeling of "pins and needles" (31%)
- Low energy (24%)
- Swelling in arms, feet, or ankles (21%)
- Dizziness (21%)
- Rash (20%)
- Arm or leg pain (19%)
- Muscle pain (18%)
- Lower respiratory tract infection (18%)
- Back pain (16%)
- High blood pressure (14%)
- Itching (10%)
Other Side Effects
- Fast heart rate
- Chest discomfort
- Shortness of breath
- Wheezing
- Ringing in the ears
- Hearing problems
- Burning sensation
- Anxiety
- Depression
Serious Side Effects
- Serious allergic reaction (anaphylaxis): trouble breathing, tightness in the throat, hives, itching, fast heartbeat, nausea, stomach pain, dizziness, lightheadedness, swelling in the face or tongue
Source: DailyMed
The following side effects have also been reported
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 or nurse immediately if any of the following side effects occur:
More common
Blurred vision
chest pain, discomfort, or tightness
confusion
cough producing mucus
difficulty breathing
difficulty in moving
dizziness
faintness or lightheadedness when getting up suddenly from a lying or sitting position
feeling unusually cold shivering
headache
hives or welts, itching, skin rash
irregular heartbeat
joint pain
muscle ache, cramps, pains, or stiffness
nervousness
pounding in the ears
redness of the skin
stomach pain
sweating
swelling of the ankles, feet, and lower legs
swollen joints
unusual tiredness or weakness
Incidence unknown
Blue lips, fingernails, or skin
chills
cloudy or bloody urine
decreased cardiac output
decreased frequency or amount of urine
difficulty in speaking
difficulty swallowing
dilated neck veins
double vision
feeling of constant movement of self or surroundings
inability to move the arms, legs, or facial muscles
increased thirst
irregular, fast, or slow, or shallow breathing
lack or loss of strength
loss of appetite
lower back or side pain
no breathing
no pulse or blood pressure
noisy breathing
pain in the joints
pain or discomfort in the arms, jaw, back, or neck
problems with muscle control or coordination
seizures
sensation of spinning
severe or sudden headache
shakiness and unsteady walk
slow or slurred speech
swollen, painful, or tender lymph glands in the neck, armpit, or groin
temporary blindness
throat tightness
trembling
trouble breathing
unconsciousness
vomiting
weakness in the arm and/or leg on one side of the body, sudden and severe
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 the throat
fear
feeling sad or empty
fever, not related to infusion
hoarseness
indigestion
irritability
loss of interest or pleasure
pain or tenderness around eyes and cheekbones
paleness of skin
sensation of change in temperature
skeletal pain
trouble concentrating
trouble sleeping
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.
Pros and cons of Fabrazyme (agalsidase beta)
Pros
Can help lower fats (GL-3) to near-normal levels in the body after just 5 months
Might be able to get the infusion at home after the first several treatments
Can be used in children as young as 2 years old
Given every 2 weeks
Cons
Can cause infusion-related side effects
Infusion can take several hours, depending on how well you tolerate the medication
No lower-cost generic available; brand-name medication only
Pharmacist tips for Fabrazyme (agalsidase beta)
Make sure to mark your appointments in your calendar to see your provider and get your Fabrazyme (agalsidase beta) infusions. Missing infusions might make the medication work less well for you.
Tell your provider if you're not feeling well, feel hot or cold, or have chest pain during the Fabrazyme (agalsidase beta) infusion. You might be having a reaction to the infusion. Your provider can adjust how fast the infusion runs or give you medications like acetaminophen (Tylenol) or diphenhydramine (Benadryl) to help relieve these symptoms.
Your provider might recommend that you take acetaminophen (Tylenol) or an antihistamine before each infusion to help prevent or lessen the severity of reactions to the Fabrazyme (agalsidase beta) infusion. These infusion reactions should also get better with time as you continue treatment with Fabrazyme (agalsidase beta).
Get help right away if you experience signs of a serious allergic reaction, including fever, chills, stomach pain, diarrhea, nausea, swelling, itching, rash, trouble breathing, and feeling agitated.
Ask your provider or infusion center if you're interested in learning about cost savings and resources for Fabrazyme (agalsidase beta). The manufacturer might be able to offer help with cost, access, and other resources for certain people who are eligible.
Frequently asked questions about Fabrazyme (agalsidase beta)
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What are the risks and warnings for Fabrazyme (agalsidase beta)?
Fabrazyme (agalsidase beta) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.
Serious allergic reaction
Risk factors: People with anti-Fabrazyme antibodies in their blood
Serious allergic reactions to Fabrazyme (agalsidase beta) are possible. These reactions can include life-threatening problems like anaphylaxis (closing of the throat and trouble breathing) and angioedema (swelling of the lips, tongue, face, or throat). The risk for severe allergic reactions might be higher in people with anti-Fabrazyme antibodies — antibodies your immune system made against the medication as an allergic response — in the blood. Tell your provider right away if you begin to have any trouble breathing, trouble swallowing, severe swelling, or hives.
Infusion-related reactions
Risk factors: People with anti-Fabrazyme antibodies in their blood
In clinical studies, over half of people who took Fabrazyme (agalsidase beta) experienced reactions during their infusions. These reactions can include fever, chills, dizziness, nausea, stomach pain, muscle pain, changes in heartbeat, or a feeling of "pins and needles," among others.
Tell your provider if you feel unwell at any point during your Fabrazyme (agalsidase beta) infusion. Your provider can adjust the infusion rate, temporarily stop the infusion, or give you medications to manage your symptoms. Call a provider if you have any reactions after your infusion as well.
If you've had an infusion reaction, your provider might give you medications to prevent reactions before your infusion at your next treatment session. Your provider might also observe you after your infusion to see how you respond to the medication in case you need appropriate treatment.
Fabrazyme (agalsidase beta) dosage forms
Typical dosing for Fabrazyme (agalsidase beta)
The dose is based on your body weight. The typical dose is 1 mg/kg of weight every 2 weeks as an infusion into your veins (intravenous or IV infusion).
Interactions between Fabrazyme (agalsidase beta) and other drugs
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.
What are alternatives to Fabrazyme (agalsidase beta)?
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References
Best studies we foundEng, C. M., et al. (2001). Safety and efficacy of recombinant human α-galactosidase A replacement therapy in Fabry’s disease. New England Journal of Medicine.
Genzyme Corporation. (2024). Fabrazyme- agalsidase beta injection, powder, lyophilized, for solution [package insert]. DailyMed.
MedlinePlus. (2022). Angioedema.
National Institute of Neurological Disorders and Stroke. (2024). Fabry disease.
National Kidney Foundation. (n.d.). Fabry disease.
National Organization for Rare Disorders. (2019). Fabry disease.
Sanofi. (2020). Long-term data on Fabrazyme® (agalsidase beta) for people with Fabry disease.
Sanofi. (2024). Prescribing information: Fabrazyme (agalsidase beta) 5mg/ 35mg powder for concentrate for solution for infusion.
Search Orphan Drug Designations and Approvals. (n.d.). Agalsidase beta. U.S. Food and Drug Administration.
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