GALAFOLD
GALAFOLD (migalastat) is an oral pharmacological chaperone developed by Amicus Therapeutics taken every other day for Fabry disease — an X-linked lysosomal storage disorder in which GLA mutations impair the alpha-galactosidase A (alpha-Gal A) enzyme, causing glycolipid accumulation in blood vessels, kidneys, heart, and nerves that leads to renal failure, cardiomyopathy, and early stroke. Rather than replacing the enzyme from outside the cell, migalastat enters cells and stabilizes specific misfolded mutant alpha-Gal A proteins, allowing them to reach the lysosome and function. GALAFOLD is approved for patients aged 12+ with amenable GLA mutations; Amicus is pursuing approval for children aged 2 to under 12.
Upcoming catalysts
Programs
Fabry Disease
Fabry disease is an X-linked lysosomal storage disorder caused by GLA mutations impairing alpha-Gal A enzyme activity, leading to glycolipid accumulation in blood vessels, kidneys, heart, and nerves that causes progressive renal failure, cardiomyopathy, and early stroke; the prior standard was IV enzyme replacement therapy every two weeks. GALAFOLD is approved for patients aged 12+ with amenable GLA mutations; the 2026 PDUFA targets label extension to children aged 2 to under 12.