Migalastat

Fabry disease is a rare X-linked lysosomal storage disorder. Traditional enzyme replacement therapy requires long-term intravenous infusion, which limits patient compliance. Migalastat, the world's first oral small-molecule protein stabilizer drug, specifically binds to the active site of α-galactosidase A, helps the misfolded enzyme restore its correct conformation and is transported to lysosomes to exert the effect of degrading substrates. It is indicated for adult Fabry disease patients and adolescent patients aged 16 and above with specific susceptibility mutations in the α-galactosidase A gene, breaking through the scenario limitation of long-term administration for rare diseases.

At present, the global market size of Fabry disease drugs has exceeded 3 billion US dollars, among which the proportion of oral small-molecule therapeutic drugs is increasing at an annual growth rate of 12%. As a representative oral drug in this field, the generic space of migalastat continues to release after its patent expiration. No generic drug has been approved in China yet, and only 1 enterprise has submitted the registration application for marketing of active pharmaceutical ingredients (APIs). The market supply side is in the early stage of competition, and no product has been included in the national centralized volume-based procurement catalog.

The original research enterprise of migalastat is Amicus Therapeutics, a US-based company, and the original research trade name is Galafold. Its core compound patents expire in 2025 and 2026 in the United States and Europe respectively, and the compound patent in China will expire in 2027. The original research dosage form is oral hard capsule with a specification of 123 mg, which has been included in the FDA Reference Listed Drug Catalog, and was approved for marketing in China in 2020 through the priority review and approval procedure. At present, no valid A-status migalastat API registration number that has been published and taken effect, except those of the declaring enterprises, can be found on the platform of the Center for Drug Evaluation of NMPA of China, and the only preparation approved for marketing in China is the original research product. (Data as of June 2025, please refer to the official CDE website for the latest information)

CATO provides a full set of migalastat impurity reference standards. The products simultaneously meet the compliance requirements of multiple regulations such as the Chinese Pharmacopoeia and FDA. Most categories support spot supply. Spot orders paid before 16:00 can be shipped on the same day, which can fully meet the reference standard use requirements in various stages of API R&D, quality research and generic drug declaration.