Drug updated on 4/17/2024
| 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).
Summary
- Elosulfase alfa (Vimizim) is indicated for the treatment of patients with Mucopolysaccharidosis type IVA, a lysosomal storage disease caused by mutations in the GALNS gene, which leads to a deficiency in N-acetylgalactosamine-6-sulfatase activity.
- A single systematic review/meta-analysis focused on the efficacy and safety of elosulfase alfa for MPS IVA included data from four cohort studies and two randomized controlled trials, covering a total of 550 participants.
- The current treatment approach for MPS IVA patients involves intravenous enzyme replacement therapy (ERT) with elosulfase alfa. This therapy has been shown to improve urinary keratan sulfate levels, physical performance tests such as walk and stair climb tests, aspects of quality of life like mobility and independence in self-care, along with respiratory function measurements.
- Although no direct comparisons were made between elosulfase alfa and other drugs, significant evidence supports its efficacy and safety in improving clinical outcomes for MPS IVA patients compared to placebo treatments.
- Given that Mucopolysaccharidosis type IVA is a rare condition with limited treatment options currently available, the systematic review underlines the importance of Vimizim as an effective therapeutic option. It emphasizes the prompt initiation of ERT after diagnosis to maximize patient outcomes, highlighting the observed benefits from using Vimizim over placebos during trials conducted so far.
- Future research should focus on evaluating the long-term impact of Vimizim usage while also comparing its effects directly against other potential treatment modalities that are currently available or being developed, thereby further solidifying its standing within management strategies used against Morquio A syndrome.
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 |