Drug updated on 9/4/2024
Dosage Form | Injection (intravenous; 20 mg/10 mL [2mg/mL]) |
Drug Class | Hydrolytic lysosomal cholesteryl ester and triacylglycerol-specific enzyme |
Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for the treatment of patients with a diagnosis of Lysosomal Acid Lipase (LAL) deficiency.
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Summary
- Kanuma (sebelipase alfa) is indicated for the treatment of patients with a diagnosis of Lysosomal Acid Lipase (LAL) deficiency.
- This summary is based on the review of one systematic review(s)/meta-analysis(es). [1]
- Sebelipase alfa treatment significantly improved serum transaminases (alanine aminotransferase and aspartate aminotransferase) and serum lipid profiles (total cholesterol, triglycerides, high-density lipoprotein cholesterol, low-density lipoprotein cholesterol) in patients with lysosomal acid lipase deficiency (LAL-D) (p < 0.01).
- No significant change was observed in gamma-glutamyl transferase (GGT) levels (p = 0.35) or liver volume (p = 0.08) with sebelipase alfa treatment.
- The study population included a broad age range, from infants (mean age 2.57 months) to adults (mean age 31.6 years), with no reported significant differences in effectiveness across these age groups.
- Adverse events related to sebelipase alfa infusions were mostly infrequent and mild-to-moderate in severity.
- No specific safety concerns or adverse effects were identified for particular population types or subgroups in the reviewed studies.
Product Monograph / Prescribing Information
Document Title | Year | Source |
---|---|---|
Kanuma (sebelipase alfa) Prescribing Information. | 2021 | 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 Practice Guidelines
Document Title | Year | Source |
---|---|---|
Sebelipase alfa for treating Wolman disease | 2024 | NICE |
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 |