Delivery 7–10 daysGalafold (migalastat) 123 mg capsules
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Description
💊 Prescription oral pharmacological chaperone for adults with a confirmed diagnosis of Fabry disease and an amenable GLA gene variant.
▪︎ Migalastat 123 mg hard capsules, carton of 14 with a punch-out dosing calendar; one capsule every other day on an empty stomach
▪︎ Manufacturer: Amicus Therapeutics
▪︎ FDA approval: August 2018
🔬 How it works: in Fabry disease a faulty GLA gene leaves cells short of alpha-galactosidase A, and globotriaosylceramide accumulates in the kidneys, heart, vessels and nerve ganglia. Enzyme replacement therapy delivers a manufactured enzyme intravenously every two weeks; migalastat delivers nothing new. With many mutations the enzyme is assembled but comes out unstable, so the cell destroys it as defective before it reaches the lysosome. Migalastat sits in the active site and holds the protein in the correct shape, letting it pass quality control and arrive at its workplace, where the acidic environment and excess substrate displace it again. 📊 Evidence: in FACETS (NEJM 2016) the primary endpoint was not met — 41% responders versus 28% on placebo (p=0.30) — because patients with non-amenable variants had been enrolled; among the amenable, left ventricular mass index fell by 7.7 g/m² over 24 months and estimated GFR changed by −0.30 mL/min/1.73 m² per year. In ATTRACT (J Med Genet 2017) 57 adults switched from infusions: renal function changed comparably, left ventricular mass index fell by 6.6 g/m² while it did not change significantly on enzyme replacement therapy.
⚠️ Prescription-only, prescribed by a specialist centre. An amenability assay of the GLA variant is mandatory before treatment — of 600 disease-causing mutations tested, 268 proved amenable, and the drug cannot work without one. It replaces enzyme replacement therapy rather than being added to it. Not studied and not recommended below an eGFR of 30 mL/min/1.73 m² or on dialysis, nor established in patients under 18. Approval was accelerated on a substrate surrogate; an effect on dialysis, infarction and stroke has not been demonstrated.
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