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 10h ago · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
GALAFOLD (migalastat hydrochloride) is an oral small molecule chaperone that stabilizes deficient alpha-galactosidase A protein in patients with Fabry disease. It binds to misfolded variants of the enzyme, allowing it to traffic to lysosomes where it breaks down accumulated glycosphingolipids. The drug is indicated for patients with amenable GLA gene variants, requiring genetic testing for patient eligibility.
$35MPart D
PeakStable
15.6 years to LOEProduct is at peak commercial maturity with modest Part D utilization, suggesting a stable but niche market; marketing and medical teams focus on variant eligibility awareness and physician engagement in rare disease networks.
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.