Sebelipase alfa

(Kanuma®)

Kanuma®

Drug updated on 5/17/2024

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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  • Sebelipase alfa (Kanuma) is an enzyme replacement therapy for individuals with Lysosomal Acid Lipase (LAL) deficiency, a rare genetic condition. This medication addresses the fundamental pathology of LAL-D by facilitating the proper breakdown and removal of cholesterol esters and triglycerides.
  • The information was derived from one systematic review/meta-analysis document that included five studies involving 110 LAL-D patients across different age groups.
  • Significant improvements were noted in serum transaminases and serum lipids among treated individuals, indicating a substantial positive effect of sebelipase alfa on these markers. However, changes in gamma-glutamyl transferase (GGT) and liver volume were not statistically significant.
  • Adverse events related to sebelipase alfa infusions were generally infrequent and ranged from mild to moderate in severity, indicating that this treatment is both safe and well-tolerated among patients with either variant of LAL-D.
  • Without specific comparative data within the reviewed studies, it's challenging to offer a direct comparison; however, given its impact on key blood markers combined with its favorable safety profile, it suggests an advancement over non-specific interventions like dietary modifications or lipid-modifying drugs previously used for managing this disease.
  • The systematic review indicates that sebelipase alfa is effective across diverse patient populations, from infants through adults, including both clinical variants of LAL-D. This broad applicability underscores the importance of enzyme replacement therapy in managing this rare genetic disorder.

Product Monograph / Prescribing Information

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

Systematic Reviews / Meta-Analyses

Clinical Practice Guidelines