Summary

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• Mepsevii (vestronidase alfa) is indicated for the treatment of Mucopolysaccharidosis VII (MPS VII, Sly syndrome) in pediatric and adult patients.
• This summary is based on the review of one randomized controlled trial(s). ^[1-2]
• Vestronidase alfa at a dose of 4 mg/kg QOW demonstrated effectiveness in reducing urinary glycosaminoglycans (GAGs) levels in patients with Mucopolysaccharidosis type VII (MPS VII), with pharmacodynamic responses indicating a plateau at this dose.
• The pharmacokinetics/pharmacodynamics (PK/PD) modeling predicts that the QOW dosing regimen results in more efficient drug delivery to GUS-deficient tissues, potentially enhancing treatment efficacy compared to less frequent dosing.
• The study population included both adult and pediatric patients with MPS VII, and no differences in effectiveness between these groups were reported, with the same dosing regimen recommended for both.
• General Safety: The study indicates that clinical evidence supports the safety of the 4 mg/kg QOW dosing regimen but does not provide specific details on adverse effects or safety outcomes.
• Significant Safety Concerns or Adverse Effects: There are no specific safety concerns or adverse effects mentioned in the study.
• There is no population types or subgroups information available in the reviewed studies.