Amicus Therapeutics is a global biotechnology company at the forefront of advanced therapies to treat a range of devastating rare and orphan diseases.
Amicus Therapeutics is currently developing migalastat, an oral drug designed to bind to and stabilize the α‐Gal A that is made in the patient’s own cells, with the intention of enabling its trafficking to lysosomes. Once delivered to lysosomes, the α‐Gal A enzyme can degrade the accumulated GL-3.
This approach is designed for patients with amenable mutations that could be capable of responding to oral migalastat as a monotherapy treatment on the basis of their genotype.
For Fabry disease patients who do not have amenable mutations, Amicus Therapeutics is investigating migalastat in combination with a novel ERT. In combination with ERT, migalastat is designed to bind to infused α‐Gal A in circulation with the intention of stabilizing the active form of the enzyme.
For more information on the development status of migalastat as a treatment option for Fabry disease, please click here.
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