.Pharmacolibrary.Drugs.A_AlimentaryTractAndMetabolism.A16A_OtherAlimentaryTractAndMetabolismProducts.A16AX14_Migalastat.Migalastat .Pharmacolibrary.Drugs.A_AlimentaryTractAndMetabolism.A16A_OtherAlimentaryTractAndMetabolismProducts.A16AX14_Migalastat.Migalastat| name: | Migalastat |
| ATC code: | A16AX14 | route: | oral |
| n-compartments | 1 |
Migalastat is an oral pharmacological chaperone used for the treatment of Fabry disease—a rare X-linked lysosomal storage disorder caused by deficiency of the enzyme alpha-galactosidase A (GLA). Migalastat stabilizes specific mutant forms of GLA, increasing their trafficking to lysosomes and thus enhancing enzymatic activity. Migalastat is approved for use in several regions including the EU, USA, and Japan.
Pharmacokinetic parameters following oral administration of migalastat 150 mg to healthy adult volunteers (mean age approx. 30 years, both sexes) under fasting conditions.
Leonowens, C, et al., & Johnson, FK (2022). Population Pharmacokinetics of Oral Migalastat in Adolescents and Adults With and Without Renal Impairment. Clinical pharmacology in drug development 11(12) 1367–1381. DOI:10.1002/cpdd.1160 PUBMED:https://pubmed.ncbi.nlm.nih.gov/36331497
McCafferty, EH, & Scott, LJ (2019). Migalastat: A Review in Fabry Disease. Drugs 79(5) 543–554. DOI:10.1007/s40265-019-01090-4 PUBMED:https://pubmed.ncbi.nlm.nih.gov/30875019
Ino, H, et al., & Hirama, T (2013). Pharmacokinetics, safety, and tolerability following single-dose migalastat hydrochloride (GR181413A/AT1001) in healthy male Japanese subjects. Journal of drug assessment 2(1) 87–93. DOI:10.3109/21556660.2013.827117 PUBMED:https://pubmed.ncbi.nlm.nih.gov/27536442