Drug updated on 12/11/2024
| Dosage Form | Injection (intravenous; 4 mg, 20 mg) |
| Drug Class | Hydrolytic lysosomal enzymes |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for treatment of non-central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients.
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Summary
- This summary is based on the review of one randomized controlled trial. [1]
- In adults with acid sphingomyelinase deficiency (ASMD), olipudase alfa led to a 22% increase in percent predicted diffusing capacity of the lung for carbon monoxide (DLCO) by week 52, compared to a 3.0% increase in the placebo group (P = .0004).
- Spleen volume decreased by 39% in the olipudase alfa group by week 52, whereas the placebo group showed a 0.5% increase (P < .0001).
- Liver volume decreased by 28% in the olipudase alfa group by week 52, compared to a 1.5% decrease in the placebo group (P < .0001).
- There were no treatment-related serious adverse events, and no adverse event-related discontinuations were reported.
- Most adverse events reported were mild.
- There is no population types or subgroups information available in the reviewed documents.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Xenpozyme (olipudase alfa-rpcp) Prescribing Information. | 2023 | Genzyme Corporation, Cambridge, MA |
Randomized Controlled Trials
| Document Title | Sex Distribution | Year | Source |
|---|---|---|---|
| A randomized, placebo-controlled clinical trial evaluating olipudase alfa enzyme replacement therapy for chronic acid sphingomyelinase deficiency (ASMD) in adults: One-year results | 36Subjects F: 61% M: 39% | 2022 | Genetics in Medicine |
Sex Distribution:
F:61%
M:39%
36Subjects
Year:
2022
Source:Genetics in Medicine