NDAORALCAPSULEPriority Review
Approved
Aug 2018
Lifecycle
Peak
Competitive Pressure
0/100
Clinical Trials
20
Data completeness
6/8 — Strong
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. First approved in 2018.
Drug data last refreshed 19h ago · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
GALAFOLD (migalastat hydrochloride) is an oral small-molecule chaperone that treats Fabry disease by stabilizing deficient alpha-galactosidase A protein, enabling it to traffic to lysosomes and break down accumulated glycosphingolipids. It is indicated for patients with amenable GLA gene variants—specific mutations where the enzyme retains partial activity but is misfolded. This precision-medicine approach requires genetic testing to identify which patients will respond.
$0.035BPart D
PeakStable
15.3 years to LOEProduct is at peak commercial performance with modest Medicare uptake (~1,121 claims in 2023); brand team likely stable in size but focused on market penetration within eligible patient population.
Mechanism 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.