GALAFOLD (migalastat hydrochloride)
Galafold treats adults with Fabry disease who possess specific genetic mutations known as amenable variants. It helps patients by reducing the accumulation of a fatty substrate called globotriaosylceramide in the kidney's capillary cells. Healthcare providers use a specialized laboratory assay to confirm that a patient’s specific genetic variant is compatible with this therapy. This approach is used for individuals whose bodies produce a version of the alpha-galactosidase A enzyme that is misfolded but still retains potential activity.
How GALAFOLD Works
The drug works by binding to the active site of the alpha-galactosidase A protein, which stabilizes the enzyme and allows it to move from the endoplasmic reticulum into the lysosome. Once inside the lysosome, the drug dissociates from the enzyme, enabling it to break down accumulated fatty substances like globotriaosylceramide (GL-3). This process restores the enzyme's ability to clear cellular waste that would otherwise build up and cause damage.
Development Insights
Details
- Status
- Prescription
- First Approved
- 2018-08-10
- Patent Cliff
- 2042
- Routes
- ORAL
- Dosage Forms
- CAPSULE
GALAFOLD Approval History
What GALAFOLD Treats
1 indicationsGALAFOLD is approved for 1 conditions since its original approval in 2018. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Fabry Disease
GALAFOLD Target & Pathway
ProTarget
GALAFOLD Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
Indication competitors
Same indication, different mechanism — what else might this patient receive?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
Drugs Similar to GALAFOLD
FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Clinical Trial Registry
3 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT01218659 results posted | AT1001-012 ATTRACT, 2010-022636-37 | Ph 3 | completed | Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease |
| NCT00925301 results posted | AT1001-011 FACETS, 2009-013459-31 | Ph 3 | completed | Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease |
| NCT01458119 AT1001-041 results posted | AT1001-041 2011-004800-40 | Ph 3 | terminated | Open-Label Phase 3 Long-Term Safety Study of Migalastat |
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
GALAFOLD FDA Label Details
Indications & Usage
FDA Label (PDF)GALAFOLD is indicated for the treatment of Fabry Disease.
GALAFOLD Patents & Exclusivity
Patents (65 active)
Pro Intelligence Preview
Deep insights for GALAFOLD
Revenue Insights
- • Quarterly revenue tracking
- • Historical trend analysis
Patent Timeline
- • Cliff: 2042
- • 66 active patents
Trial Analysis
- • 3 total trials
- • Stage: Declining
Competitive Landscape
- • 2 similar drugs
- • Same target/indication analysis
Full approval history • All patents • Revenue trends • Competitor analysis
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment