Galafold (migalastat)
An approved treatment for Fabry Disease.
The same compound appears under different names depending on the context. Here is how to identify Migalastat wherever you encounter it, plus the key facts at a glance.
- Generic name
- Migalastat
- Brand name
- Galafold
- Development codes
- AT1001, DGJ
- Drug class
- Pharmacological chaperone
- Manufacturer
- Amicus Therapeutics (acquired by BioMarin in April 2026)
- How it's taken
- Taken as a single oral capsule (123 mg) every other day.
The first oral treatment for Fabry disease. Rather than replacing the missing enzyme, migalastat stabilizes the patient's own misfolded enzyme so it can work properly. Only effective for patients with specific 'amenable' genetic mutations.
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Where Migalastat fits
First oral therapy for Fabry disease, offering an alternative to biweekly IV enzyme replacement. Only effective for patients with specific amenable GLA mutations, which must be confirmed before starting therapy.
How Migalastat works
In some Fabry patients, the body makes alpha-galactosidase A enzyme, but it's misfolded and gets destroyed before it can do its job. Migalastat acts as a molecular chaperone, binding to the misfolded enzyme and helping it fold into the correct shape. Once properly folded, the enzyme is released to do its normal job of breaking down GL-3.
Mechanism: Pharmacological chaperone that stabilizes the patient's own alpha-galactosidase A enzyme
Side effects and safety
Common side effects include headache, nasopharyngitis, urinary tract infection, fever, and nausea. Generally well-tolerated compared to IV enzyme replacement. Not effective for all GLA mutations, only 'amenable' variants.
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 Migalastat
Taken as a single oral capsule (123 mg) every other day. Must be taken on an empty stomach, with no food or caffeine for 2 hours before and 2 hours after the dose. The every-other-day schedule is important for proper enzyme cycling.
Availability and cost
Only available as the brand-name product.
First oral pharmacological chaperone for Fabry disease. Small molecule that stabilizes the patient's own mutant enzyme, but only works for patients with specific amenable GLA mutations.
Help paying for Galafold
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
Eligible commercially insured patients may pay as little as $0 per month. Program maximums apply; not for Medicare, Medicaid, TRICARE or VA.
For: private insurance · source - Free medicine program
The Amicus Patient Assistance Program provides free product to eligible uninsured or underinsured patients.
For: no insurance, underinsured · source - Insurance and case manager help
Case Managers verify and explain insurance coverage, help with prior authorization and coordinate at-home deliveries.
For: private insurance, Medicare, Medicaid, TRICARE, no insurance, underinsured · source
Good to know: Your doctor enrolls you with the Patient Referral Form. Spanish-speaking case managers available.
- 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…”
Access and eligibility
Galafold is approved only for adults with Fabry disease, and it only works for patients with specific 'amenable' GLA gene variants. There are hundreds of approved amenable variants. Your doctor can check whether your specific mutation is amenable using the manufacturer's online tool before prescribing.
Check eligibility toolSource: Galafold.com (Amicus Therapeutics)
Access program details are provided for informational purposes and may vary based on insurance coverage, geographic location, and individual circumstances. Confirm current eligibility directly with the manufacturer or your specialty pharmacy.
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 | 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 You are here | 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
In the Phase 3 FACETS trial, the share of patients with at least a 50% drop in kidney GL-3 at 6 months was 52% on migalastat versus 45% on placebo overall, with a larger difference in patients who started with higher GL-3 levels; the US approval is an accelerated approval based on this GL-3 reduction. The current US label lists more than 350 amenable GLA variants. Non-inferiority to ERT demonstrated in the ATTRACT study.
Development history
Approved by FDA in August 2018 for adults with Fabry disease who have an amenable GLA variant. Represented a paradigm shift as the first oral therapy for Fabry disease and the first pharmacological chaperone for a lysosomal storage disorder.
Explore Fabry Disease trials
Other Fabry Disease treatments
Common questions about Migalastat
▸What is Migalastat (Galafold)?
The first oral treatment for Fabry disease. Rather than replacing the missing enzyme, migalastat stabilizes the patient's own misfolded enzyme so it can work properly. Only effective for patients with specific 'amenable' genetic mutations.
▸How does Migalastat work?
In some Fabry patients, the body makes alpha-galactosidase A enzyme, but it's misfolded and gets destroyed before it can do its job. Migalastat acts as a molecular chaperone, binding to the misfolded enzyme and helping it fold into the correct shape. Once properly folded, the enzyme is released to do its normal job of breaking down GL-3.
▸What are the side effects of Migalastat?
Common side effects include headache, nasopharyngitis, urinary tract infection, fever, and nausea. Generally well-tolerated compared to IV enzyme replacement. Not effective for all GLA mutations, only 'amenable' variants.
▸How is Migalastat taken?
Taken as a single oral capsule (123 mg) every other day. Must be taken on an empty stomach, with no food or caffeine for 2 hours before and 2 hours after the dose. The every-other-day schedule is important for proper enzyme cycling.
▸Is Migalastat FDA approved?
Yes, Migalastat (Galafold) is FDA approved (2018) for the treatment of Fabry Disease.
▸How do I know if my Fabry mutation is amenable to migalastat?
Migalastat only works for specific GLA gene variants. Amicus Therapeutics provides an online amenability checking tool where your doctor can verify if your specific mutation responds to the drug. The current US label lists more than 350 amenable variants.
▸Why is migalastat taken every other day instead of daily?
The every-other-day dosing schedule is designed for proper enzyme cycling. Migalastat binds to the enzyme and helps it fold correctly, then the drug needs to clear so the stabilized enzyme can be released to do its work. Daily dosing could actually reduce effectiveness.
▸What dietary restrictions apply to migalastat?
Migalastat must be taken on an empty stomach, with no food or caffeine for 2 hours before and 2 hours after each dose. Water, clear fruit juice without pulp and caffeine-free soft drinks are allowed. Food can interfere with the drug's absorption and effectiveness.
▸Can I switch from Fabrazyme to Galafold?
Patients with amenable mutations can switch from IV enzyme replacement to oral migalastat. The ATTRACT study demonstrated non-inferiority when switching from ERT to migalastat. Your doctor will confirm your mutation is amenable before switching.
▸What was the significance of migalastat's approval?
Approved in August 2018, migalastat was the first oral therapy for Fabry disease and the first pharmacological chaperone for any lysosomal storage disorder, representing a paradigm shift from IV enzyme replacement to oral disease management.
▸What patient support is available for Galafold?
Amicus Assist provides case management, insurance navigation, copay support, and financial assistance for eligible patients taking Galafold.