Fabrazyme (agalsidase beta)
An approved treatment for Fabry Disease.
The same compound appears under different names depending on the context. Here is how to identify Agalsidase beta wherever you encounter it, plus the key facts at a glance.
- Generic name
- Agalsidase beta
- Brand name
- Fabrazyme
- Drug class
- Enzyme replacement therapy
- Manufacturer
- Sanofi Genzyme
- How it's taken
- Given as an intravenous infusion every 2 weeks at 1 mg/kg.
The standard enzyme replacement therapy for Fabry disease, FDA-approved for adults and children 2 years and older with confirmed Fabry disease. Provides the alpha-galactosidase A enzyme that patients' bodies can't make enough of, clearing the fatty substance (GL-3) that accumulates in cells and causes organ damage.
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Where Agalsidase beta fits
Standard enzyme replacement therapy for Fabry disease, providing the missing alpha-galactosidase A enzyme. Typically first-line for classic Fabry disease patients, especially males with severe manifestations.
How Agalsidase beta works
In Fabry disease, a genetic defect means the body produces little or no alpha-galactosidase A enzyme. Without this enzyme, a fatty substance called GL-3 builds up in blood vessel walls, kidneys, heart, and nerves, causing progressive damage. Fabrazyme provides a manufactured version of the missing enzyme, which enters cells and breaks down the accumulated GL-3.
Mechanism: Enzyme replacement therapy providing functional alpha-galactosidase A enzyme
Side effects and safety
Infusion reactions are common, including fever, chills, headache, tingling, and nausea. Some patients develop antibodies that may reduce effectiveness. Fabrazyme has a boxed warning for life-threatening allergic reactions, including anaphylaxis, which can happen early in treatment or after years of infusions, so treatment is started in a healthcare setting with emergency equipment ready. Pre-medication can help reduce infusion reactions. People with advanced Fabry disease whose heart function is weakened may be at higher risk of serious complications from infusion reactions and should be watched closely during infusions.
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Agalsidase beta
Given as an intravenous infusion every 2 weeks at 1 mg/kg. Infusions typically take 1.5-4.5 hours. Usually administered at an infusion center, though home infusion may be arranged after initial doses.
Availability and cost
Only available as the brand-name product.
Enzyme replacement therapy produced through recombinant DNA technology in mammalian cell culture. Biweekly IV infusions of a biologic enzyme for a rare lysosomal storage disorder.
Help paying for Fabrazyme
Pick your insurance to see which help fits. Drugmaker copay cards can't be used with Medicare, Medicaid or TRICARE; charity funds are the usual route there.
- Copay help
CareConnectPSS Copay Assistance Program helps with Fabrazyme copays, coinsurance and deductibles up to a program maximum. Excludes Medicare, Medicaid, VA, DoD, TRICARE.
For: private insurance · source - Infusion cost help
The copay program also covers certain infusion-related costs (mixing, administration, supplies). Not covered for residents of MA or RI.
For: private insurance · source - Free medicine program
Patient Assistance Program may provide Fabrazyme to patients with no insurance or whose insurance denied coverage; apply with your doctor.
For: no insurance, underinsured · source - Insurance and case manager help
Case Manager helps with insurance, out-of-pocket costs and claims; Patient Education Liaisons offer disease education.
The official page does not say who qualifies. Ask the program. · source
Good to know: Call 1-800-745-4447, option 3. Sanofi describes the free-drug program as a temporary solution until coverage is secured. The copay program does not cover office visits, labs, premedications or travel.
- From a charity · NORD RareCareFabry Disease Medical Assistance fundOpen
Pays for: Medical and medication costs.
The foundation says: “Accepting Applications” - From a charity · NORD RareCareFabry Disease Premium Copay Assistance fundOpen
Pays for: Insurance premiums and copays.
The foundation says: “Accepting Applications” - From a charity · TotalAssist (formerly PAN Foundation)Fabry Disease fundOpen
Pays for: Out-of-pocket costs for approved medications, up to $9,000 per year. Requires health insurance (any kind).
- From a charity · Fabry Support & Information GroupFabry Assist fundApply directly
Pays for: One-time help with past-due Fabry-related medical bills, non-pharmacy medical items (cooling vests, compression stockings, hearing aids), Fabry testing and limited travel.
The foundation says: “Status not shown on page” - From a charity · The Assistance FundFabry Disease fundWaitlist
Pays for: Copays, coinsurance, deductibles and other health-related expenses.
The foundation says: “WAITLIST — Accepting Waitlist Patients. TAF is currently accepting requests to join the enrollment waitlist for this program. Waitlists a…”
How Fabry disease treatments compare
Three FDA-approved treatments are available for Fabry disease: two enzyme replacement therapies and one oral pharmacological chaperone. Your Fabry disease specialist (a geneticist, cardiologist, or nephrologist experienced with Fabry) will recommend a treatment based on your specific GLA mutation, disease severity, organ involvement, and personal preferences. Galafold is only an option if your mutation is classified as amenable.
3 FDA-approved fabry disease treatments are available: Fabrazyme (agalsidase beta, approved 2003); Galafold (migalastat, approved 2018); Elfabrio (pegunigalsidase alfa, approved 2023). Fabrazyme is typically used as standard first-line ert; 20+ years of clinical experience.
| Drug | How it works | How it’s given | How often | Where you get it | Typical use | FDA approved |
|---|---|---|---|---|---|---|
Fabrazyme agalsidase beta You are here | Enzyme replacement — provides the alpha-galactosidase A enzyme your body can't make enough of, clearing GL-3 buildup from cells | Intravenous infusion (1 mg/kg) | Every 2 weeks | Infusion center or home infusion | Standard first-line ERT; 20+ years of clinical experience | 2003 |
Galafold migalastat | Pharmacological chaperone — stabilizes your body's own misfolded enzyme so it can fold correctly and function (only works for amenable GLA mutations) | Oral capsule (123 mg) on an empty stomach | Every other day (not on consecutive days) | Home (oral, no infusion needed) | Alternative to ERT for patients with amenable GLA mutations | 2018 |
Elfabrio pegunigalsidase alfa | PEGylated enzyme replacement — same enzyme as Fabrazyme with PEG molecules attached for longer circulation and potentially better tissue penetration | Intravenous infusion (1 mg/kg) | Every 2 weeks | Infusion center or home infusion | Next-generation ERT; can shorten infusion time to 1.5 hours once established | 2023 |
This chart summarizes approved Fabry disease treatments to help you understand the landscape. It is not medical advice. Galafold only works for patients with specific amenable GLA gene mutations and must be confirmed by genetic testing before prescribing. Treatment decisions depend on your specific mutation, disease severity, organ involvement, and personal preferences. Always discuss options with your Fabry disease specialist, which may include a geneticist, cardiologist, or nephrologist depending on your primary organ involvement.
Clinical trial results
Demonstrated 69% clearance of GL-3 from kidney blood vessel cells. Long-term data shows stabilization of kidney function and reduction in cardiac events when started early.
Development history
Approved by FDA in April 2003 as the first enzyme replacement therapy for Fabry disease. Has over 20 years of clinical experience and remains the most widely used treatment for Fabry disease worldwide.
Explore Fabry Disease trials
Other Fabry Disease treatments
Common questions about Agalsidase beta
▸What is Agalsidase beta (Fabrazyme)?
The standard enzyme replacement therapy for Fabry disease, FDA-approved for adults and children 2 years and older with confirmed Fabry disease. Provides the alpha-galactosidase A enzyme that patients' bodies can't make enough of, clearing the fatty substance (GL-3) that accumulates in cells and causes organ damage.
▸How does Agalsidase beta work?
In Fabry disease, a genetic defect means the body produces little or no alpha-galactosidase A enzyme. Without this enzyme, a fatty substance called GL-3 builds up in blood vessel walls, kidneys, heart, and nerves, causing progressive damage. Fabrazyme provides a manufactured version of the missing enzyme, which enters cells and breaks down the accumulated GL-3.
▸What are the side effects of Agalsidase beta?
Infusion reactions are common, including fever, chills, headache, tingling, and nausea. Some patients develop antibodies that may reduce effectiveness. Fabrazyme has a boxed warning for life-threatening allergic reactions, including anaphylaxis, which can happen early in treatment or after years of infusions, so treatment is started in a healthcare setting with emergency equipment ready. Pre-medication can help reduce infusion reactions. People with advanced Fabry disease whose heart function is weakened may be at higher risk of serious complications from infusion reactions and should be watched closely during infusions.
▸How is Agalsidase beta taken?
Given as an intravenous infusion every 2 weeks at 1 mg/kg. Infusions typically take 1.5-4.5 hours. Usually administered at an infusion center, though home infusion may be arranged after initial doses.
▸Is Agalsidase beta FDA approved?
Yes, Agalsidase beta (Fabrazyme) is FDA approved (2003) for the treatment of Fabry Disease.
▸How does Fabrazyme compare to the oral treatment Galafold?
Fabrazyme (agalsidase beta) is an IV enzyme replacement given every 2 weeks that is approved for people 2 years and older with confirmed Fabry disease, whatever their mutation, while Galafold (migalastat) is an oral chaperone taken every other day that only works for patients with specific amenable GLA mutations. Fabrazyme replaces the missing enzyme directly; Galafold stabilizes the patient's own misfolded enzyme.
▸What are infusion reactions with Fabrazyme and how are they managed?
Infusion reactions including fever, chills, headache, tingling, and nausea are common with Fabrazyme. Pre-medication with antihistamines, acetaminophen, or corticosteroids can help reduce these reactions. Slowing the infusion rate may also help.
▸Can Fabrazyme be given at home?
After initial doses at an infusion center to monitor for reactions, home infusion may be arranged for stable patients. Each infusion typically takes 1.5-4.5 hours and is given every 2 weeks at 1 mg/kg body weight.
▸Do antibodies affect Fabrazyme's effectiveness?
Some patients develop antibodies to agalsidase beta that may reduce its effectiveness over time. Antibody levels are typically monitored during treatment, and clinical response is assessed to determine if therapy adjustments are needed.
▸How long has Fabrazyme been available?
Fabrazyme was approved by the FDA in April 2003 as the first enzyme replacement therapy for Fabry disease and has over 20 years of clinical experience. It remains the most widely used treatment for Fabry disease worldwide.
▸How does Fabrazyme compare to Elfabrio?
Both are IV enzyme replacement therapies for Fabry disease, but Elfabrio (pegunigalsidase alfa) is PEGylated for a potentially longer half-life. Elfabrio was approved in 2023 as a newer alternative, while Fabrazyme has over 20 years of safety and efficacy data.