Product Description
Migalastat (Galafold™), a pharmacological chaperone, stabilizes and facilitates trafficking of amenable mutant forms of alpha-galactosidase A enzyme from the endoplasmic reticulum to lysosomes and increases its lysosomal activity. Oral migalastat is the first pharmacological chaperone approved for treating patients [aged ≥ 18 years (USA and Canada) or ≥ 16 years in other countries] with Fabry disease who have a migalastat-amenable GLA mutation. (Sourced from: https://pubmed.ncbi.nlm.nih.gov/30875019/)
Mechanisms of Action: GH Agonist
Novel Mechanism: No
Modality: Small Molecule
Route of Administration: Oral
FDA Designation: None *
Approval Status: Approved
Approved Countries: Argentina | Australia | Austria | Belgium | Brazil | Canada | Chile | Colombia | Croatia | Cyprus | Czech | Denmark | Estonia | European Medicines Agency | Finland | France | Germany | Greece | Hong Kong | Hungary | Iceland | Ireland | Israel | Italy | Japan | Korea | Latvia | Lithuania | Luxembourg | Netherlands | Norway | Poland | Portugal | Romania | Slovakia | Slovenia | Spain | Sweden | Switzerland | Taiwan | Turkey | United Kingdom | United States
Approved Indications: None
Known Adverse Events: None
Company: Amicus
Company Location: PHILADELPHIA PA 19104
Company CEO: Bradley L. Campbell
Additonal Commercial Interests: None
Clinical Description
Countries in Clinic: Australia, France, Japan, Portugal, Spain, United Kingdom, United States
Active Clinical Trial Count: 4
Highest Development Phases
Phase 3: Fabry Disease|Kidney Diseases
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
|---|---|---|---|---|---|
| AT1001-025 | P3 |
Unknown Status |
Fabry Disease|Kidney Diseases |
2026-12-13 |
|
| jRCT2051220125 | P3 |
Recruiting |
Fabry Disease |
2025-12-31 |
|
| AT1001-036 | P3 |
Active, not recruiting |
Fabry Disease |
2025-12-01 |
|
| AT1001-025 | P3 |
Recruiting |
Kidney Diseases|Fabry Disease |
2024-12-31 |