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Fabry Disease Treatments Compared

Name: Fabry Disease Treatments Compared
Creator: Jason Hunter
Published: 2026-10-09

3 FDA-approved drugs and 1 in development, side by side from the current prescribing labels. Newest label Aug 2025. Checked October 9, 2026.

Enzyme replacement infusionsFabrazyme · Elfabrio

Chaperone pill (amenable variants only)Galafold

Three drugs are FDA-approved for Fabry disease. Fabrazyme (agalsidase beta) and Elfabrio (pegunigalsidase alfa) are enzyme replacement infusions given every 2 weeks, while Galafold (migalastat) is a capsule taken every other day that is approved only for people whose GLA gene variant is amenable to it. Fabrazyme is the only one approved for children, from age 2; Elfabrio and Galafold are for adults. Elfabrio is the only drug tested head to head, against Fabrazyme in people already taking it, and kidney function declined at nearly the same rate on both over 2 years. Both infusions carry a boxed warning for severe allergic reactions including anaphylaxis; Galafold has no boxed warning, holds accelerated approval and is not recommended with severe kidney impairment.

Side by side

3 approved Fabry disease treatments at a glance

How each drug is given, how often, who it is approved for and when it was first approved, from the current FDA label.

Fabry Disease treatments compared: route, schedule, approved use and approval date
Drug	How it is given	How often	Approved for	First approved
Fabrazyme agalsidase beta Sanofi (Genzyme)	IV infusion every 2 weeks, started in a healthcare settingAbout 90 minutes per dose	1 mg/kg by IV infusion every 2 weeks, starting at 0.25 mg per minute and sped up as tolerated to a minimum of 1.5 hours	Adults and children 2 and older with confirmed Fabry disease	April 24, 2003
Elfabrio pegunigalsidase alfa Chiesi (developed with Protalix)	IV infusion every 2 weeksAbout 90 minutes per dose	1 mg/kg by IV infusion every 2 weeks; the first 4 to 6 infusions run at set rates by body weight, then can be shortened to a minimum of 1.5 hours	Adults with confirmed Fabry disease	May 9, 2023
Galafold migalastat Amicus Therapeutics (BioMarin)	1 capsule every other day by mouth, on an empty stomach	123 mg capsule every other day at the same time of day, with no food or caffeine for 2 hours before and 2 hours after	Adults with confirmed Fabry disease and an amenable GLA gene variant, under accelerated approval	August 10, 2018

The calendar

A year on each Fabry disease treatment

Every dose in a year, drawn to the label schedule, so you can see at a glance how often each one is given.

Fabrazyme

Infusion, at a clinic

26 doses a year

Elfabrio

Infusion, at a clinic

26 doses a year

Galafold

By mouth, at home

183 doses a year

Counts follow the label schedule for a full year on the maintenance dose. Drugs given in cycles or adjusted to blood tests have no fixed count, so their schedule is written out instead.

The evidence

What the pivotal Fabry disease trials showed

The primary result from the trial each FDA approval rests on, as reported on the label.

Before you compare

How to read the Fabry disease trial results

1
Your gene variant decides if Galafold is an option
Galafold only helps when the person's own faulty enzyme can be steadied by the drug, which a lab test of the GLA variant decides. People in its trial with non-amenable variants saw no change. Fabrazyme and Elfabrio work regardless of variant.
2
Only Elfabrio and Fabrazyme were compared directly
The Elfabrio trial switched 77 adults who had been on Fabrazyme for an average of 5.7 years to either Elfabrio or continued Fabrazyme for 2 years. Kidney function fell 2.4 points a year on Elfabrio and 2.3 on Fabrazyme. The trial says nothing about people starting treatment for the first time.
3
The kidney deposit results are not a ranking
Fabrazyme's trial counted people whose small kidney blood vessels were cleared of GL-3 deposits at 20 weeks (69% against 0% on placebo). Galafold's counted people with at least a 50% drop in kidney GL-3 at 6 months (52% against 45% on placebo, 71% against 44% in men). Different scoring and different patients mean the numbers do not compare.
4
Infusions take time and start in a clinic
Both infusions run at least 1.5 hours once a person is used to them, and the first ones run slower. Fabrazyme must be started in a healthcare setting with emergency equipment, and Elfabrio's label calls for emergency equipment to be on hand during every infusion.

Fabry Disease pivotal trial results by drug
Drug	Pivotal trial	Primary result
Fabrazyme agalsidase beta Sanofi (Genzyme)	Study 1, 58 people aged 16 to 61 who had never had enzyme replacement	20 of 29 (69%) on Fabrazyme cleared GL-3 deposits from small kidney blood vessels at 20 weeks vs 0 of 29 on placebo
Elfabrio pegunigalsidase alfa Chiesi (developed with Protalix)	BALANCE (Trial 2), 77 adults already on Fabrazyme for at least 1 year, Elfabrio vs Fabrazyme for 2 years Trial record or paper	Kidney function (eGFR) declined 2.4 mL/min per year on Elfabrio and 2.3 on Fabrazyme, a difference of 0.1 (95% CI -2.3 to 2.1)
Galafold migalastat Amicus Therapeutics (BioMarin)	FACETS (Study 1), 67 adults, of whom 50 had amenable variants Trial record or paper	52% on Galafold vs 45% on placebo had at least a 50% drop in kidney GL-3 at 6 months (13 of 25 vs 9 of 20); in men, 71% vs 44%

Safety

Fabry Disease treatment side effects and warnings

Fabrazyme and Elfabrio carry a boxed warning, the FDA's strongest. The rest list their main precautions.

Fabry Disease treatments: most common side effects and key warnings from the FDA labels
Drug	Most common side effects	Key warning
Fabrazyme agalsidase beta Sanofi (Genzyme)	infusion reactions 59%, upper respiratory infection 53%, chills 49% and fever 39%	Boxed warning Boxed warning for life-threatening allergic reactions including anaphylaxis. Start in a healthcare setting with emergency equipment; premedication with antihistamines, fever reducers or steroids may be used.
Elfabrio pegunigalsidase alfa Chiesi (developed with Protalix)	infusion reactions 32%, nasopharyngitis 21%, headache 21% and diarrhea 19%	Boxed warning Boxed warning for allergic reactions including anaphylaxis. The label also warns about membranoproliferative glomerulonephritis, a kidney inflammation reported in 1 patient; kidney tests should be monitored.
Galafold migalastat Amicus Therapeutics (BioMarin)	headache 35%, nasopharyngitis 18%, urinary tract infection 15% and nausea 12%	No boxed warning and no warnings section on the label. Only for amenable GLA variants; not recommended with severe kidney impairment (eGFR below 30) or dialysis.

Not yet approved

Fabry disease treatments in Phase 3 or under FDA review

Investigational drugs with a Phase 3 trial, shown separately because their results are not from a label. Status as of the date in each row.

Fabry Disease drugs in development
Drug	Status	How it works and is given	Pivotal trial	Result so far	What is next
venglustat Sanofi	Phase 3as of October 9, 2026	Oral pill that slows production of the fatty substance that builds up in Fabry disease (substrate reduction)Tablet by mouth once a day	CARAT, Phase 3, 104 adults with thickened heart muscle, vs usual carePrimary endpoint: Change in left ventricular mass index on cardiac MRI ClinicalTrials.gov	Not yet reported. A separate Phase 3, PERIDOT (122 people), missed its pain endpoint because pain fell in both groups (Sanofi, February 2, 2026)	Primary completion May 2026; results not yet announced Sanofi press release, February 2, 2026

Cost

Help paying for Fabry disease treatment

Drugmaker copay and free-drug programs, plus charity funds for the condition, checked every week.

Questions

Fabry Disease treatment questions people ask

Fabrazyme vs Elfabrio: what is the difference for Fabry disease?

Both replace the missing alpha-galactosidase A enzyme by IV infusion every 2 weeks at 1 mg/kg. Elfabrio is a newer version of the enzyme, made in plant cells and linked to PEG, a coating used to change how a drug behaves in the body.

Who it is for: Fabrazyme covers adults and children 2 and older. Elfabrio covers adults only.

Trial results: Elfabrio is the only Fabry drug tested head to head. In 77 adults already on Fabrazyme, kidney function declined 2.4 mL/min per year on Elfabrio and 2.3 on Fabrazyme over 2 years, a difference of 0.1.

Safety: both carry a boxed warning for anaphylaxis. Elfabrio's label also warns about membranoproliferative glomerulonephritis, a kidney inflammation reported in 1 trial patient, and calls for regular kidney tests. Infusion reactions were reported in 32% of people on each drug in the head-to-head trial.

Switching: Elfabrio's label includes starting infusion rates for people coming from another enzyme replacement therapy. Whether to switch is a decision for the treating doctor.

More: Fabrazyme drug page and Elfabrio drug page

Source: FDA label for Fabrazyme, DailyMed, effective August 2025; FDA label for Elfabrio, DailyMed, effective July 2024

Who can take Galafold for Fabry disease?

Adults with confirmed Fabry disease and a GLA gene variant that a lab assay shows is amenable to migalastat. The label recommends a clinical genetics professional review the variant when its meaning is uncertain. Galafold is not recommended for people with severe kidney impairment (eGFR below 30) or on dialysis, and it is not approved for children. It is taken as 1 capsule every other day with no food or caffeine for 2 hours before and after.

Is there a pill for Fabry disease?

Yes, Galafold (migalastat), but only for adults with an amenable GLA variant. Two other pills are in trials: venglustat from Sanofi, whose Phase 3 heart trial CARAT has finished enrolling, and lucerastat from Idorsia, whose new Phase 3 trial Fab-Klear (NCT07778667) is not yet recruiting. Venglustat's other Phase 3 Fabry trial, PERIDOT, missed its pain goal in February 2026.

Which Fabry disease treatment is approved for children?

Only Fabrazyme, approved from age 2 since March 2021. Elfabrio and Galafold are approved for adults. Both have pediatric studies recruiting: Elfabrio in children (NCT06328608) and Galafold in children aged 2 to under 12 (NCT06904261).

Are there new Fabry disease treatments in clinical trials?

As of October 2026, the furthest along is venglustat, an oral drug from Sanofi, whose Phase 3 CARAT trial in 104 adults with thickened heart muscle finished its main data collection in May 2026 with results not yet announced. Lucerastat, an oral drug from Idorsia, has a Phase 3 trial (NCT07778667) that is not yet recruiting. Gene therapies 4D-310 from 4D Molecular Therapeutics (NCT05629559) and AMT-191 from uniQure (NCT06270316) are in Phase 1/2 trials that are recruiting.

Does Fabrazyme slow kidney decline in Fabry disease?

Its label reports 2 kinds of evidence. In a trial of 82 people with more advanced disease, kidney, heart or stroke events or death occurred in 28% on Fabrazyme against 42% on placebo, a difference that was not statistically certain. In an observational comparison of 122 treated people with matched untreated patients, kidney function declined 1.5 mL/min per year on Fabrazyme against 3.2 untreated.

Go deeper

More on Fabry Disease at Trial Friend

Sources

Where every number on this page comes from

Approved-drug facts are from the current FDA prescribing information on DailyMed, with the label's effective date. Investigational-drug facts are from ClinicalTrials.gov and the sources named in each row.

Fabrazyme (agalsidase beta) prescribing information, Sanofi (Genzyme), label dated Aug 2025. DailyMed
Elfabrio (pegunigalsidase alfa) prescribing information, Chiesi (developed with Protalix), label dated Jul 2024. DailyMed. Pivotal trial: ClinicalTrials.gov
Galafold (migalastat) prescribing information, Amicus Therapeutics (BioMarin), label dated Aug 2025. DailyMed. Pivotal trial: ClinicalTrials.gov
venglustat, Sanofi: ClinicalTrials.gov record; Sanofi press release, February 2, 2026. Checked October 9, 2026.

This page compares what the FDA labels and pivotal trials report. It is not medical advice, and no trial on this page compared these drugs with each other. Written and checked by Jason Hunter; the method is on the editorial standards page.