Summary

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• 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.