Formulary
Migalastat: Indications, Dosing, Side Effects and Interactions
Migalastat is a pharmacological chaperone that binds to the active sites of certain mutant forms of alpha-galactosidase A, thereby stabilising these mutant...
MedNext Academy | 3 min read
Migalastat: Indications, Dosing, Side Effects and Interactions
Migalastat is a pharmacological chaperone that binds to the active sites of certain mutant forms of alpha-galactosidase A, thereby stabilising these mutant...
Drug action
Migalastat is a pharmacological chaperone that binds to the active sites of certain mutant forms of alpha-galactosidase A, thereby stabilising these mutant forms in the endoplasmic reticulum, and facilitating normal trafficking to lysosomes.
Indications and dose
**Fabry's disease (under expert supervision)**
- **Adult** (By Mouth): 123 mg once daily on alternate days, to be taken at the same time of day.
**Fabry's disease (under expert supervision) for migalastat**
- **Child 12-17 years (body-weight 45 kg and above)** (By mouth): 123 mg once daily on alternate days, to be taken at the same time of day.
Side effects
Common or very common Constipation; defaecation urgency; depression; diarrhoea; dizziness; dry mouth; dyspnoea; epistaxis; fatigue; gastrointestinal discomfort; headache; muscle complaints; nausea; pain; palpitations; proteinuria; sensation abnormal; skin reactions; torticollis; vertigo; weight increased
Interactions
**Other interactions (5):**
- Based upon in vitro data, migalastat is not an inducer of CYP1A2, 2B6, or 3A4.
- Furthermore, migalastat is not an inhibitor or a substrate of CYP1A2, 2A6, 2B6, 2C8, 2C9, 2C19, 2D6, 2E1, or 3A4/5.
- Migalastat is not a substrate for MDR1 or BCRP, nor is it an inhibitor of BCRP, MDR1, or BSEP human efflux transporters.
- In addition, migalastat is not a substrate for MATE1, MATE2-K, OAT1, OAT3, or OCT2, nor is it an inhibitor of OATP1B1, OATP1B3, OAT1, OAT3, OCT1, OCT2, MATE1, or MATE2-K human uptake transporters.
- Effect of other drugs on migalastat Co-administration of migalastat with caffeine decreases migalastat systemic exposure (AUC and C max ) which may reduce Galafold efficacy (see section 5.2).
Pregnancy
Manufacturer advises avoid-toxicity in animal studies.
Breast feeding
Manufacturer advises avoid-present in milk in animal studies.
Renal impairment
Prescribing in renal impairment . Manufacturer advises avoid if eGFR less than 30 mL/minute/1.73 m 2 .
Medicinal forms
Clinical governance
**Author:** MedNext Clinical Team. **Clinical reviewer:** Dr Shameer Deen, MBBS, MS, MRCS. **Sources:** BNF, Indian Pharmacopoeia, CIMS India. **Correction:** Report errors at support@mednext.academy.
Inside MedNext for this topic
- 411 MedNext-authored chapters
- 80,000+ MCQ bank
- 15 study modes
- Growing visual cheat sheets
Study modes
- Notes
- MCQ
- Audio
- Video
- Visual
- 3D Anatomy
- Trace
- Flashcards
- Mnemonics
- Image Bank
- Clinical
- Microscopy
- Audio QBank
- Cadaver
- Book Match
Continue reading
Subject HubPharmacology Hub
All pharmacology resources in one place.
Drug SchedulesIndian Drug Schedules
Schedule H, H1, X and G classifications.
FormularyBrowse all drugs
Search 1,850+ drug profiles.
Study Migalastat in the MedNext app
Challenge yourself with targeted pharmacology MCQs on this drug class. Every explanation links back to the chapter that teaches the concept.
Start drug challengeExplore the full formulary

