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Vestronidase alfa

(Mepsevii®)

Mepsevii®

Drug updated on 3/28/2024

Dosage FormInjection (intravenous: 10 mg/5 mL)
Drug ClassRecombinant human lysosomal beta glucuronidases
Ongoing and
Completed Studies		ClinicalTrials.gov

Indication

  • For the treatment of Mucopolysaccharidosis VII (MPS VII, Sly syndrome) in pediatric and adult patients.

Summary
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  • Vestronidase alfa (Mepsevii) is an enzyme replacement therapy used for the treatment of Mucopolysaccharidosis VII (MPS VII, Sly syndrome) in pediatric and adult patients.
  • The information provided was derived from one document which was a Randomized Controlled Trial.
  • In this trial, twelve subjects aged between 8-25 years completed a Phase 3 study receiving vestronidase alfa at doses of 4mg/kg intravenously over periods ranging from 24 to 48 weeks.
  • All participants showed significantly reduced urinary glycosaminoglycans levels and clinical improvement as per multi-domain responder index after completion of the blind-start study followed by an extension study lasting up to additional 144 weeks.
  • The safety profile remained consistent throughout both studies with most adverse events being mild to moderate in severity; no discontinuations due to adverse events were reported and there were no significant changes observed in standard safety chemistry panel results during the course of these trials.
  • Despite seven out of eleven subjects testing positive for neutralizing antibodies against vestronidase alfa, all continued showing reduction in urinary GAG levels indicating that antibody formation did not affect drug efficacy or cause infusion associated reactions; thus demonstrating long-term safety and durability of clinical efficacy with continuous use of vestronidase alfa (Mepsevii).

Product Monograph / Prescribing Information

Document TitleYearSource
Mepsevii (vestronidase alfa) Prescribing Information.2020Ultragenyx Pharmaceutical Inc, Novato, CA

Randomized Controlled Trials

Document TitleSex DistributionYearSource
The long-term safety and efficacy of vestronidase alfa, rhGUS enzyme replacement therapy, in subjects with mucopolysaccharidosis VII.
12Subjects
F: 67%
M: 33%
2020Molecular genetics and metabolism

Document Title

The long-term safety and efficacy of vestronidase alfa, rhGUS enzyme replacement therapy, in subjects with mucopolysaccharidosis VII.

Sex Distribution:

F:67%
M:33%
12Subjects

Year:

2020

Source:Molecular genetics and metabolism