Medical genetics
Fabry disease: the approved drugs compared for prescribers
Elfabrio, Fabrazyme, Galafold side by side on what each first fill waits on, what the label requires you to monitor and what an insurer asks to see, with FDA decisions, label changes and open trials for Fabry disease.
When to suspect Fabry disease and what to test →The 3 Fabry disease prescribing guides
One page per drug with the dispensing gate, prior authorization documentation, dosing, monitoring and interactions, each line sourced.
The drugs side by side
Each cell is a summary of that drug's prescribing guide, which carries the full detail and a source on every line. Insurer thresholds are one public policy shown as an example; plans differ.
| Elfabriopegunigalsidase alfa-iwxj | Fabrazymeagalsidase beta | Galafoldmigalastat | |
|---|---|---|---|
| Label dated | July 8, 2024 | August 22, 2025 | August 28, 2025 |
| Starting dose | 1mg/kg actual body weight IV every 2 weeks. Medical support including CPR equipment readily available. | 1mg/kg actual body weight IV every 2 weeks. | 123mg orally once every other day at the same time of day; not on consecutive days; swallow whole, do not cut, crush or chew. No titration and no other strength. |
| Dispensing gate | No REMS. Prior authorization, Elfabrio Start Form, a site with CPR-capable medical support for initial infusions, and premedication planned case by case | No REMS. Boxed warning: initiate in a healthcare setting with monitoring and CPR equipment. Prior authorization and a site of care before the first infusion | No REMS; Galafold Referral Form (also a prescription in some states) with patient consent to Amicus Assist by fax 1-833-264-2873 or email assist@amicusrx.com; specialty pharmacy ships after any prior authorization |
| Required monitoring | Medical support including CPR equipment at every infusion; serum creatinine and UPCR (no schedule given); IgG and IgE ADA after hypersensitivity or IARs; lyso-Gb3 when switching with pre-existing ADA; close monitoring if cardiac function is compromised | Supervised infusion with anaphylaxis readiness at every dose; consider premedication; close monitoring if cardiac function is compromised; consider IgE testing after a severe reaction; no scheduled labs | No scheduled monitoring in the label; not recommended at eGFR below 30 or on dialysis; caffeine within 2 hours of a dose lowers exposure |
| Example PA threshold | FEP example: age 18 or older, confirmed Fabry disease, prescriber agrees to monitor for hypersensitivity during infusion; 2-year approval | Cigna example: documented low alpha-Gal A activity in leukocytes or fibroblasts, or a pathogenic GLA variant; specialist prescriber; up to 1mg/kg every 2 weeks; no concurrent Galafold or Elfabrio | Cigna example: age 18 or older, documented pathogenic or likely pathogenic amenable GLA variant per in vitro assay, geneticist, nephrologist or Fabry specialist, no concurrent Fabrazyme or Elfabrio |
| Support line | Chiesi Total Care 1-800-784-5840 (Monday to Friday 8 AM to 8 PM ET), fax 1-877-435-2524; as little as $0 for drug and for infusion administration on eligible commercial plans; no infusion copay or infusion assistance in MA or RI | CareConnectPSS Case Managers 1-800-745-4447, option 3 (Monday to Friday 8 AM to 6 PM ET); Co-Pay Program 1-855-448-6520 for eligible commercial plans; Patient Assistance Program for uninsured or denied patients | Amicus Assist, 1-833-264-2872 (1-833-AMICUS-A), Monday to Friday 8 AM to 8 PM ET; Case Manager contacts office and patient within 1 business day; copay as little as $0 for commercial plans; Amicus Patient Assistance Program |
- Label dated
- July 8, 2024
- Starting dose
- 1mg/kg actual body weight IV every 2 weeks. Medical support including CPR equipment readily available.
- Dispensing gate
- No REMS. Prior authorization, Elfabrio Start Form, a site with CPR-capable medical support for initial infusions, and premedication planned case by case
- Required monitoring
- Medical support including CPR equipment at every infusion; serum creatinine and UPCR (no schedule given); IgG and IgE ADA after hypersensitivity or IARs; lyso-Gb3 when switching with pre-existing ADA; close monitoring if cardiac function is compromised
- Example PA threshold
- FEP example: age 18 or older, confirmed Fabry disease, prescriber agrees to monitor for hypersensitivity during infusion; 2-year approval
- Support line
- Chiesi Total Care 1-800-784-5840 (Monday to Friday 8 AM to 8 PM ET), fax 1-877-435-2524; as little as $0 for drug and for infusion administration on eligible commercial plans; no infusion copay or infusion assistance in MA or RI
- Label dated
- August 22, 2025
- Starting dose
- 1mg/kg actual body weight IV every 2 weeks.
- Dispensing gate
- No REMS. Boxed warning: initiate in a healthcare setting with monitoring and CPR equipment. Prior authorization and a site of care before the first infusion
- Required monitoring
- Supervised infusion with anaphylaxis readiness at every dose; consider premedication; close monitoring if cardiac function is compromised; consider IgE testing after a severe reaction; no scheduled labs
- Example PA threshold
- Cigna example: documented low alpha-Gal A activity in leukocytes or fibroblasts, or a pathogenic GLA variant; specialist prescriber; up to 1mg/kg every 2 weeks; no concurrent Galafold or Elfabrio
- Support line
- CareConnectPSS Case Managers 1-800-745-4447, option 3 (Monday to Friday 8 AM to 6 PM ET); Co-Pay Program 1-855-448-6520 for eligible commercial plans; Patient Assistance Program for uninsured or denied patients
- Label dated
- August 28, 2025
- Starting dose
- 123mg orally once every other day at the same time of day; not on consecutive days; swallow whole, do not cut, crush or chew. No titration and no other strength.
- Dispensing gate
- No REMS; Galafold Referral Form (also a prescription in some states) with patient consent to Amicus Assist by fax 1-833-264-2873 or email assist@amicusrx.com; specialty pharmacy ships after any prior authorization
- Required monitoring
- No scheduled monitoring in the label; not recommended at eGFR below 30 or on dialysis; caffeine within 2 hours of a dose lowers exposure
- Example PA threshold
- Cigna example: age 18 or older, documented pathogenic or likely pathogenic amenable GLA variant per in vitro assay, geneticist, nephrologist or Fabry specialist, no concurrent Fabrazyme or Elfabrio
- Support line
- Amicus Assist, 1-833-264-2872 (1-833-AMICUS-A), Monday to Friday 8 AM to 8 PM ET; Case Manager contacts office and patient within 1 business day; copay as little as $0 for commercial plans; Amicus Patient Assistance Program
FDA decisions for Fabry disease
From the Trial Friend FDA decision calendar, which records each date from the sponsor's or the FDA's own announcement.
- Date not disclosedST-920 (isaralgagene civaparvovec), BLA (rolling), PTC TherapeuticsPending
Recent label changes
What changed in each label revision, from the FDA's approval letters and the labels' own revision lists.
- August 2025Galafold. The current DailyMed version took effect on August 28th, with no new prescribing text. Its wording matches the March 2025 label, and the outer carton panel carries the August date. March 2025 label
- August 2025Fabrazyme. DailyMed posted a new version with small wording changes and a product listing update. No dose, warning or use changed. Its recent changes list shows the month as XX/2024, an apparent placeholder. FDA label
- March 2025Galafold. The FDA approved adding angioedema, a swelling under the skin, to the side effects reported after approval. The FDA approval letter for that change is dated March 19th. FDA letter S-009
- July 2024Fabrazyme. The FDA added a boxed warning for severe allergic reactions, including anaphylaxis, as part of a change for enzyme replacement therapies as a group. Fabrazyme must now be started in a healthcare setting with emergency equipment. FDA letter
- June 2024Galafold. The FDA approved adding new amenable GLA variants to the label's table on June 5th. FDA letter S-008
- May 2024Elfabrio. The label added a smaller 5mg vial alongside the 20mg vial and corrected the trial registry number for the head-to-head trial. The dose, infusion rates and warnings did not change. FDA label
- February 2024Fabrazyme. The warnings gained advice to consider antihistamines, fever reducers or steroids before infusions. The pregnancy and breastfeeding sections were rewritten using information Genzyme collected after approval. FDA letter
- June 2023Galafold. On June 20th the FDA approved adding caffeine to the fasting rule and naming which drinks are allowed. On the same day it approved one new amenable variant for the table. FDA letter S-006, S-007
Open trials
Questions prescribers ask about Fabry disease
Which Fabry disease drugs require a REMS?
None of the 3 Fabry disease drugs on this page has a REMS: Elfabrio, Fabrazyme and Galafold. Each prescribing guide lists what does gate the first fill.
What monitoring do Fabry disease drugs require?
Elfabrio: Medical support including CPR equipment at every infusion; serum creatinine and UPCR (no schedule given); IgG and IgE ADA after hypersensitivity or IARs; lyso-Gb3 when switching with pre-existing ADA; close monitoring if cardiac function is compromised. Fabrazyme: Supervised infusion with anaphylaxis readiness at every dose; consider premedication; close monitoring if cardiac function is compromised; consider IgE testing after a severe reaction; no scheduled labs. Galafold: No scheduled monitoring in the label; not recommended at eGFR below 30 or on dialysis; caffeine within 2 hours of a dose lowers exposure.
What do insurers require before covering Elfabrio, Fabrazyme and Galafold?
Each answer below is one public insurer policy, an example rather than every plan. Elfabrio: FEP example: age 18 or older, confirmed Fabry disease, prescriber agrees to monitor for hypersensitivity during infusion; 2-year approval. Fabrazyme: Cigna example: documented low alpha-Gal A activity in leukocytes or fibroblasts, or a pathogenic GLA variant; specialist prescriber; up to 1mg/kg every 2 weeks; no concurrent Galafold or Elfabrio. Galafold: Cigna example: age 18 or older, documented pathogenic or likely pathogenic amenable GLA variant per in vitro assay, geneticist, nephrologist or Fabry specialist, no concurrent Fabrazyme or Elfabrio.
Which support programs start Fabry disease patients on therapy?
Elfabrio: Chiesi Total Care 1-800-784-5840 (Monday to Friday 8 AM to 8 PM ET), fax 1-877-435-2524; as little as $0 for drug and for infusion administration on eligible commercial plans; no infusion copay or infusion assistance in MA or RI. Fabrazyme: CareConnectPSS Case Managers 1-800-745-4447, option 3 (Monday to Friday 8 AM to 6 PM ET); Co-Pay Program 1-855-448-6520 for eligible commercial plans; Patient Assistance Program for uninsured or denied patients. Galafold: Amicus Assist, 1-833-264-2872 (1-833-AMICUS-A), Monday to Friday 8 AM to 8 PM ET; Case Manager contacts office and patient within 1 business day; copay as little as $0 for commercial plans; Amicus Patient Assistance Program.
A reference for licensed professionals, checked on November 7, 2026. Labels and plan policies change; the label on DailyMed and the plan's current policy govern. Not medical advice. For patients: the Fabry disease page and the first 90 days pages for Elfabrio, Fabrazyme, Galafold.