.Pharmacolibrary.Drugs.A_AlimentaryTractAndMetabolism.A16A_OtherAlimentaryTractAndMetabolismProducts.A16AB18_VestronidaseAlfa.VestronidaseAlfa .Pharmacolibrary.Drugs.A_AlimentaryTractAndMetabolism.A16A_OtherAlimentaryTractAndMetabolismProducts.A16AB18_VestronidaseAlfa.VestronidaseAlfa| name: | VestronidaseAlfa |
| ATC code: | A16AB18 | route: | intravenous |
| n-compartments | 2 |
Vestronidase alfa is a recombinant human beta-glucuronidase enzyme replacement therapy used to treat mucopolysaccharidosis VII (MPS VII, Sly syndrome), an ultra-rare genetic disorder caused by deficiency of the beta-glucuronidase enzyme. It is FDA and EMA approved for use in both pediatric and adult patients.
Population pharmacokinetic parameters in pediatric and adult patients with MPS VII following intravenous infusion of vestronidase alfa.
Qi, Y, et al., & Shi, J (2019). Pharmacokinetic and Pharmacodynamic Modeling to Optimize the Dose of Vestronidase Alfa, an Enzyme Replacement Therapy for Treatment of Patients with Mucopolysaccharidosis Type VII: Results from Three Trials. Clinical pharmacokinetics 58(5) 673–683. DOI:10.1007/s40262-018-0721-y PUBMED:https://pubmed.ncbi.nlm.nih.gov/30467742