Drug updated on 5/17/2024
Dosage Form | Injection (intravenous; 10 mg/5 mL) |
Drug Class | Recombinant human lysosomal beta glucuronidases |
Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for the treatment of Mucopolysaccharidosis VII (MPS VII, Sly syndrome) in pediatric and adult patients.
Latest News
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Summary
- 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 Title | Year | Source |
---|---|---|
Mepsevii (vestronidase alfa) Prescribing Information. | 2020 | Ultragenyx Pharmaceutical Inc., Novato, CA |
Randomized Controlled Trials
Document Title | Sex Distribution | Year | Source |
---|---|---|---|
The long-term safety and efficacy of vestronidase alfa, rhGUS enzyme replacement therapy, in subjects with mucopolysaccharidosis VII. | 12Subjects F: 67% M: 33% | 2020 | Molecular genetics and metabolism |
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. | Data not availableSubjects F: null% M: null% | 2019 | Clinical Pharmacokinetics |
Sex Distribution:
F:67%
M:33%
12Subjects
Year:
2020
Source:Molecular genetics and metabolism
Sex Distribution:
![No Data](/img/Nodata_Icon.png)
Year:
2019
Source:Clinical Pharmacokinetics