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Drug

Migalastat hydrochloride

Approved · EMA

Late-stage development

Also known as migalastat (as hydrochloride), migalastat HCl.

2

Active clinical trials

1

Regulatory events

Latest activity

beta

Recent clinical, regulatory, research and industry developments relating to this drug.

An Open-label Study to Evaluate the Safety and Pharmacokinetics of Migalastat HCl in Subjects With Fabry Disease and Amenable GLA Variants and Severe Renal Impairment or End-Stage Renal Disease Treated With Hemodialysis

Clinical trial2026-07-06Results expected Q4 2026 · ClinicalTrials.gov

An Open-label Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of 12 Month Treatment With Migalastat in Pediatric Subjects (Aged 2 to < 12 Years) With Fabry Disease and Amenable GLA Variants

Clinical trial2026-06-25Results expected Q4 2027 · ClinicalTrials.gov

A Long-term, Open-label Study to Evaluate the Safety, Pharmacodynamics, and Efficacy of Migalastat in Subjects > 12 Years of Age With Fabry Disease and Amenable GLA Variants

Clinical trial2026-02-05Results posted · ClinicalTrials.gov

An Open-label Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of 12 Month Treatment With Migalastat in Pediatric Subjects (Aged 12 to <18 Years) With Fabry Disease and Amenable GLA Variants

Clinical trial2021-11-30Results posted · ClinicalTrials.gov

An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects With Fabry Disease

Clinical trial2020-12-21Results posted · ClinicalTrials.gov

An Open-label Phase 2A Study to Investigate Drug-Drug Interactions Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Subjects With Fabry Disease

Clinical trial2018-12-19Results posted · ClinicalTrials.gov

A Randomized, Open-Label Study to Compare the Efficacy and Safety of AT1001 and Enzyme Replacement Therapy (ERT) in Patients With Fabry Disease and AT1001-Responsive GLA Mutations, Who Were Previously Treated With ERT

Clinical trial2018-11-01Results posted · ClinicalTrials.gov

A Phase 2, Open-Label, Single Dose Level, 12-Week Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of AT1001 in Patients With Fabry Disease

Clinical trial2018-10-31Results posted · ClinicalTrials.gov

A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety and Pharmacodynamics of AT1001 in Patients With Fabry Disease and AT1001-Responsive GLA Mutations

Clinical trial2018-10-30Results posted · ClinicalTrials.gov

A Phase 2, Open-Label, Multicenter, 12-Week Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AT1001 in Patients With Fabry Disease

Clinical trial2018-10-30Results posted · ClinicalTrials.gov

A Phase 2, Open-Label, Multiple Dose Level, 12-Week Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of AT1001 in Female Patients With Fabry Disease

Clinical trial2018-10-03Results posted · ClinicalTrials.gov

Approval: Galafold (EMA)

Regulatory2016-05-25EMA

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name

Migalastat hydrochloride

Aliases & brands

migalastat (as hydrochloride)migalastat HCl

RxNorm CUI

2054264

Regulatory jurisdictions

ema

Regulatory timeline

1 event

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Earliest approval

2016-05-25

Latest approval

2016-05-25

Authorities

EMA

Total events

1

emaEuropean Medicines Agency· 1 event

2016-05-25Approval

Approval: Galafold (EMA)

Indication: Galafold is indicated for long-term treatment of adults and adolescents aged 12 years and older with a confirmed diagnosis of Fabry disease (α-galactosidase A deficiency) and who… Show full indication

Galafold is indicated for long-term treatment of adults and adolescents aged 12 years and older with a confirmed diagnosis of Fabry disease (α-galactosidase A deficiency) and who have an amenable mutation (see the tables in section 5.1).

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.

Clinical trials

15 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme

CLINICALTRIALS.GOV · LIVE REGISTRY

15

registered trials across all phases

LATEST COMPLETION 2024

2

Active studies

1

Recruiting

8

Late-stage (III+)

11

Completed

2

Discontinued

PHASE DISTRIBUTIONn = 15

Phase 11Phase 26Phase 38

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

An Open-label Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of 12 Month Treatment With Migalastat in Pediatric Subjects (Aged 2 to < 12 Years) With Fabry Disease and Amenable GLA Variants

RecruitingPH IIIAmicus Therapeuticsn=82028

An Open-label Study to Evaluate the Safety and Pharmacokinetics of Migalastat HCl in Subjects With Fabry Disease and Amenable GLA Variants and Severe Renal Impairment or End-Stage Renal Disease Treated With Hemodialysis

Active, not recruitingPH IIIAmicus Therapeuticsn=142026

A Long-term, Open-label Study to Evaluate the Safety, Pharmacodynamics, and Efficacy of Migalastat in Subjects > 12 Years of Age With Fabry Disease and Amenable GLA Variants

CompletedPH IIIAmicus Therapeuticsn=162024

An Open-label Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of 12 Month Treatment With Migalastat in Pediatric Subjects (Aged 12 to <18 Years) With Fabry Disease and Amenable GLA Variants

CompletedPH IIIAmicus Therapeuticsn=222021

An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects With Fabry Disease

CompletedPH IIIAmicus Therapeuticsn=842019

Recent completions

Trials that read out recently, adding to the completed evidence base.

A Long-term, Open-label Study to Evaluate the Safety, Pharmacodynamics, and Efficacy of Migalastat in Subjects > 12 Years of Age With Fabry Disease and Amenable GLA Variants

CompletedPH IIIAmicus Therapeuticsn=162024

An Open-label Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of 12 Month Treatment With Migalastat in Pediatric Subjects (Aged 12 to <18 Years) With Fabry Disease and Amenable GLA Variants

CompletedPH IIIAmicus Therapeuticsn=222021

An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects With Fabry Disease

CompletedPH IIIAmicus Therapeuticsn=842019

A Randomized, Open-Label Study to Compare the Efficacy and Safety of AT1001 and Enzyme Replacement Therapy (ERT) in Patients With Fabry Disease and AT1001-Responsive GLA Mutations, Who Were Previously Treated With ERT

CompletedPH IIIAmicus Therapeuticsn=682015

A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Single Dose Escalation Trial to Evaluate the Safety, Tolerability and Pharmacokinetics of Migalastat Hydrochloride Given Intravenously to Healthy Volunteers With an Open-Label, Randomized, Two-Way Crossover Arm

CompletedPH IAmicus Therapeuticsn=312014

A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety and Pharmacodynamics of AT1001 in Patients With Fabry Disease and AT1001-Responsive GLA Mutations

CompletedPH IIIAmicus Therapeuticsn=672014

References & data sources

RxNorm (U.S. National Library of Medicine) — drug identity
ClinicalTrials.gov — clinical trials
Regulatory event sources are credited in the Regulatory Timeline above.