Sebelipase alfa: first global approval

Drugs. 2015 Nov;75(16):1935-40. doi: 10.1007/s40265-015-0479-6.


Sebelipase alfa (Kanuma™) is a recombinant human lysosomal acid lipase (LAL) developed by Synageva BioPharma Corp. (now Alexion Pharmaceuticals, Inc.) for long-term enzyme replacement therapy in patients with LAL deficiency. The agent, administered by intravenous infusion once weekly or once every other week, acts to replace the deficient enzyme activity in patients with LAL deficiency, reducing lysosomal lipid accumulation, and thereby improving disease-related abnormalities such as dyslipidaemia and liver abnormalities. Sebelipase alfa received its first global approval, in the EU, in August 2015 for long-term enzyme replacement therapy in patients of all ages with LAL deficiency. Regulatory submissions have also been filed in the USA, Mexico and Japan for use in this indication. This article summarizes the milestones in the development of sebelipase alfa leading to this first approval for the treatment of LAL deficiency.

Publication types

  • Review

MeSH terms

  • Drug Approval*
  • Enzyme Replacement Therapy*
  • Humans
  • Internationality*
  • Patents as Topic
  • Recombinant Proteins / adverse effects
  • Recombinant Proteins / pharmacokinetics
  • Recombinant Proteins / pharmacology
  • Recombinant Proteins / therapeutic use
  • Sterol Esterase / adverse effects
  • Sterol Esterase / pharmacokinetics
  • Sterol Esterase / pharmacology
  • Sterol Esterase / therapeutic use*
  • Wolman Disease / drug therapy*


  • Recombinant Proteins
  • Sterol Esterase
  • Sebelipase alfa

Supplementary concepts

  • Lysosomal acid lipase deficiency