NDAORALCAPSULEPriority Review
Approved
Aug 2018
Lifecycle
Peak
Competitive Pressure
0/100
Clinical Trials
24
Data completeness
7/8 — Comprehensive
Priority Review
GALAFOLD (migalastat hydrochloride) by Amicus Therapeutics is alpha-galactosidase a (alpha-gal a) protein (encoded by the galactosidase alpha gene, gla ), which is deficient in fabry disease. Approved for fabry disease. First approved in 2018.
Drug data last refreshed 20h ago · AI intelligence enriched 3w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
GALAFOLD (migalastat hydrochloride) is an oral capsule approved in August 2018 for Fabry disease in patients with amenable galactosidase alpha gene (GLA) variants. It functions as a pharmacological chaperone that stabilizes misfolded alpha-galactosidase A (alpha-Gal A) protein, enabling its trafficking from the endoplasmic reticulum to lysosomes where it can break down accumulated glycosphingolipids (GL-3 and lyso-Gb3). The drug represents a precision medicine approach, requiring genetic testing to confirm GLA variant amenability before treatment. GALAFOLD is positioned as a disease-modifying option for eligible Fabry disease patients with specific genetic mutations.
$0.0BPart D
15.7 years to LOEMechanism of Action
alpha-galactosidase A (alpha-Gal A) protein (encoded by the galactosidase alpha gene, GLA ), which is deficient in Fabry disease. This binding stabilizes alpha-Gal A allowing its trafficking from the endoplasmic reticulum into the lysosome where it exerts its action. In the lysosome, at a lower pH…
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.