Vestronidase alfa


Vestronidase alfa

Drug updated on 5/17/2024

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


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

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  • Vestronidase alfa (Mepsevii) is indicated for the treatment of Mucopolysaccharidosis VII (MPS VII, Sly syndrome) in pediatric and adult patients. This treatment has shown significant reductions in urinary glycosaminoglycans levels and improvements in clinical outcomes such as pulmonary function, motor function, range of motion, mobility, visual acuity, and a sustained reduction in fatigue.
  • Two randomized controlled studies were reviewed to gather information about Mepsevii. These studies provided insights into Mepsevii's safety profile and effectiveness but did not offer direct comparative data with other treatments.
  • Both studies emphasize the safety profile of vestronidase alfa. Most adverse events reported were mild to moderate with no instances of treatment or study discontinuation due to these events. No noteworthy changes were observed in standard safety chemistry panel indicating its safe use.
  • The presence of anti-drug antibodies, including neutralizing antibodies, was noted among some subjects during the trial period; however, they did not correlate with infusion-related reactions, suggesting that the immune response does not significantly impact treatment safety or efficacy.
  • A dosing regimen of 4 mg/kg QOW (once every other week) based on pharmacokinetic modeling was found efficient, delivering the drug leading to reduced urinary GAGs from baseline. This indicates increased efficiency towards affected tissues, thus suggesting superior efficacy at the recommended dosage frequency.
  • Subjects ranging from 8 years old up to 25 years old participated, demonstrating applicability across a wide age range. Considering body weight as a significant covariate for pharmacokinetic modeling suggests a tailored approach accounting for variability among different age groups, ensuring optimal delivery effect both for pediatric and adult populations.

Product Monograph / Prescribing Information

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

Randomized Controlled Trials

Clinical Practice Guidelines