Drug updated on 9/4/2024
Dosage Form | Injection (intravenous; 5 mg/5 mL [1 mg/mL]) |
Drug Class | Hydrolytic lysosomal enzymes |
Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for treatment for patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome).
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Summary
- Vimizim (elosulfase alfa) is indicated for the treatment of patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome).
- This summary is based on the review of one systematic review(s)/meta-analysis(es). [1]
- Urinary Keratan Sulfate (uKS): The ERT group showed a reduction in uKS levels compared to the placebo group, with a mean difference of -0.260, indicating improvement in the biochemical marker for MPS IVA.
- Physical Performance: The ERT group demonstrated slight improvements in the 6-Minute Walk Test (mean difference: -0.102), 3-Minute Stair Climb Test (mean difference: -0.182), and lung function measures including Forced Vital Capacity (mean difference: -0.587) and FEV1 (mean difference: -0.293).
- Quality of Life and Mobility: Patients on ERT experienced improvements in self-care abilities (mean difference: -0.360), reduced need for caregiver assistance (mean difference: -0.408), and enhanced maximal voluntary ventilation (mean difference: -0.311).
- There is no safety information available in the reviewed studies.
- There is no population types or subgroups information available in the reviewed studies.
Product Monograph / Prescribing Information
Document Title | Year | Source |
---|---|---|
Vimizim (elosulfase alfa) prescribing information. | 2019 | BioMarin Pharmaceutical Inc., Novato, CA |
Systematic Reviews / Meta-Analyses
Document Title | Year | Source |
---|---|---|
Intravenous elosulfase alfa for mucopolysaccharidosis type IVA: A systematic review and meta-analysis. | 2022 | Journal of Personalized Medicine |
Clinical Practice Guidelines
Document Title | Year | Source |
---|---|---|
Consensus statement on enzyme replacement therapy for mucopolysaccharidosis IVA in Central and South-Eastern European countries. | 2022 | Orphanet Journal of Rare Diseases |
Recommendations for the management of MPS IVA: Systematic evidence- and consensus-based guidance. | 2019 | Orphanet Journal of Rare Diseases |