Drug updated on 3/28/2024
Dosage Form | Injection (intravenous: 10 mg/5 mL) |
Drug Class | Recombinant 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
- 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 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 |
Sex Distribution:
F:67%
M:33%
12Subjects
Year:
2020
Source:Molecular genetics and metabolism