Sebelipase alfa

(Kanuma®)

Sebelipase alfa

Drug updated on 9/4/2024

Dosage FormInjection (intravenous; 20 mg/10 mL [2mg/mL])
Drug ClassHydrolytic 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
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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.

Product Monograph / Prescribing Information

Document TitleYearSource
Kanuma (sebelipase alfa) Prescribing Information.2021Alexion Pharmaceuticals Inc., New Haven, CT

Systematic Reviews / Meta-Analyses

Clinical Practice Guidelines