Drug updated on 7/25/2024
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
- Sebelipase alfa (Kanuma) is indicated for the treatment of patients diagnosed with Lysosomal Acid Lipase (LAL) deficiency, a rare genetic disorder caused by mutations in the LIPA gene.
- The information was derived from one systematic review/meta-analysis document that evaluated seven records across five individual studies involving 110 LAL-D patients.
- Significant improvements were observed in serum transaminases and lipid profiles, including total cholesterol, triglycerides, HDL-C, and LDL-C. No significant changes were noted in gamma-glutamyl transferase (GGT) or liver volume.
- Adverse events related to sebelipase alfa infusions were mostly mild to moderate in severity.
- Before sebelipase alfa's availability, treatments included dietary modifications and some lipid-modifying drugs which primarily controlled symptoms rather than addressing the underlying cause of LAL-D.
- The study population ranged from infants to adults with mean ages spanning from 2.57 months to 31.6 years; however, efficacy by specific subgroups such as disease severity or genetic variations was not detailed.
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 |