Drug updated on 4/17/2024
| Dosage Form | Injection (intravenous; 20 mg/10 mL) |
| Drug Class | Hydrolytic lysosomal enzymes |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- For the treatment of patients with a diagnosis of Lysosomal Acid Lipase (LAL) deficiency
Summary
- Sebelipase alfa (Kanuma) is used for the treatment of patients with a diagnosis of Lysosomal Acid Lipase (LAL) deficiency.
- The systematic review and meta-analysis, consisting of one document, evaluates the safety and efficacy of sebelipase alfa in treating LAL-D.
- This analysis included seven records from five individual studies involving 110 patients diagnosed with LAL-D. The age range among these participants was between 2.57 months to 31.6 years.
- Treatment outcomes were measured based on changes in serum transaminases, serum lipids, gamma-glutamyl transferase levels and liver volume from baseline to end-of-treatment period.
- Significant improvements were observed in both serum transaminases and other lipid parameters after using sebelipase alfa. However, no significant differences were noted for GGT levels or liver volume post-treatment.
- Adverse events related to infusions during treatment with sebelipase alfa were mostly mild-to-moderate in severity and occurred infrequently indicating that it is safe as well as effective for treating both variants of LAL-D.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Kanuma (sebelipase alfa) Prescribing Information. | 2022 | Alexion Pharmaceuticals Inc., New Haven, CT |
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 review report: sebelipase alfa (Kanuma). | 2018 | CADTH |