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Pharmacological chaperone (α-galactosidase A) Pregnancy: US label 8.1: Only three pregnant women with Fabry disease were exposed in clinical trials, so available data are insufficient to assess drug-associated risks of major birth defects, miscarriage or adverse maternal or fetal outcomes. In animal reproduction studies no adverse developmental effects were observed in rats and rabbits during organogenesis at up to 26 and 54 times the recommended dose based on AUC. A pregnancy data-collection study exists for women with Fabry disease.

Migalastat

Brand names: Galafold

Migalastat is an oral pharmacological chaperone used for the long-term treatment of Fabry disease in patients who have an amenable galactosidase alfa (GLA) gene mutation.

Auto-extracted from the source labelling — not yet independently clinician-verified. These values were distilled from the UK SPC (or the US label where noted) but have not had a clinician sign-off. Confirm against the current SmPC before prescribing.

Adult dose

Dose: 123 mg once every other day
Route: Oral (capsule swallowed whole - do not cut, crush or chew), on an empty stomach
Frequency: Once every other day, at the same time of day; do not take on consecutive days
US label 2.2 (GALAFOLD): 'The recommended dosage of GALAFOLD is 123 mg orally once every other day.' Patient selection (2.1): only adults with confirmed Fabry disease who have an amenable GLA variant; treatment is indicated where the amenable GLA variant is interpreted by a clinical genetics professional as causing Fabry disease (pathogenic, likely pathogenic) in the patient's clinical context - consultation with a clinical genetics professional is strongly recommended where the variant is of uncertain significance or may be benign. Administration: take on an empty stomach - no food or caffeine for at least 2 hours before and 2 hours after the dose, giving a minimum 4 hour fast; water (plain, flavoured or sweetened), pulp-free fruit juices and caffeine-free carbonated beverages may be consumed during the fast. Missed dose (2.3): take the missed dose if within 12 hours of the scheduled time; if more than 12 hours have passed, take the next dose on the next planned dosing day, keeping the original every-other-day schedule. Paediatric: safety and effectiveness have not been established in paediatric patients (8.4). US labelling - verify against UK labelling.

Dose auto-extracted from US FDA prescribing information (openFDA / DailyMed) — cross-check; US labelling may differ from UK — not yet clinician-verified. Always confirm against the product SmPC and your local formulary before prescribing.

Contraindications

  • None stated (US label section 4: 'None.')

Side effects

  • Headache
  • Nasopharyngitis
  • Urinary tract infection
  • Nausea
  • Pyrexia

Interactions

  • Caffeine decreases migalastat AUC and Cmax, which may reduce efficacy - avoid caffeine for at least 2 hours before and 2 hours after the dose

Clinical monograph

How it works

It is a pharmacological chaperone that selectively and reversibly binds to amenable mutant forms of alpha-galactosidase A, stabilising the enzyme and facilitating its trafficking to lysosomes to restore activity.

Prescribing in practice

  • It is only effective in patients with an amenable mutation, so genotype must be confirmed against the list of amenable mutations before and during use.
  • It is taken orally on alternate days on an empty stomach, with food avoided for a period before and after dosing to ensure absorption.
  • It is an alternative to enzyme replacement therapy and is not used concurrently with it.

Monitoring

Monitor renal and cardiac parameters and overall disease progression as part of long-term Fabry disease care.

Counselling the patient

  • Take on alternate days and avoid food for the specified periods before and after each dose.
  • Take the dose at around the same time on dosing days for consistent absorption.
  • Continue regular specialist follow-up to monitor your Fabry disease.

Evidence & guidelines

Migalastat is recommended for Fabry disease with amenable mutations, supported by trials showing maintenance of renal function comparable to enzyme replacement therapy.

Reference: NICE TA517; SmPC; Drug verified in RxNorm (NLM); confirm dosing against the manufacturer SPC (eMC). Verify against your local formulary and current prescribing references before prescribing. The structured dose values shown have been reviewed by a clinician. Monograph status: clinician-reviewed (2026-07-04).

Related

Curated clinical cross-links plus same-class fallbacks.

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