Drug updated on 12/11/2024
| Dosage Form | Injection (intravenous; 20 mg/10 mL [2mg/mL]) |
| Drug Class | Hydrolytic lysosomal cholesteryl ester-specific enzymes |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for the treatment of patients with a diagnosis of Lysosomal Acid Lipase (LAL) deficiency.
Latest News
Summary
- This summary is based on the review of one systematic review/meta-analysis. [1]
- Sebelipase alfa significantly improved serum transaminase levels (p < 0.01) and lipid profiles, including total cholesterol, triglycerides, High-Density Lipoprotein (HDL), and Low-Density Lipoprotein (LDL) cholesterol (p < 0.01) in patients with lysosomal acid lipase deficiency (LAL-D), demonstrating a strong positive effect on these markers.
- No statistically significant changes were observed in gamma-glutamyl transferase (GGT) levels (p = 0.35) or liver volume (p = 0.08), indicating that these measures were unaffected by sebelipase alfa treatment in this patient population.
- Adverse events related to sebelipase alfa infusions were generally infrequent and mild-to-moderate in severity, with no serious infusion-related reactions reported in the reviewed studies.
- There were no specific safety concerns or adverse effects highlighted for particular population types or subgroups within the study data.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Kanuma (sebelipase alfa) Prescribing Information. | 2024 | Alexion Pharmaceuticals, Inc., Boston, MA |
Systematic Reviews / Meta-Analyses
| Document Title | Year | Source |
|---|---|---|
| Enzyme replacement therapy in lysosomal acid lipase deficiency (LAL-D): a systematic literature review | 2021 | Therapeutic Advances in Rare Disease |
Clinical Practice Guidelines
| Document Title | Year | Source |
|---|---|---|
| Sebelipase alfa for treating Wolman disease | 2024 | National Institute for Health and Care Excellence |
| Initial assessment and ongoing monitoring of lysosomal acid lipase deficiency in children and adults: Consensus recommendations from an international collaborative working group. | 2020 | Molecular Genetics and Metabolism |