Sebelipase alfa - Alexion Pharmaceuticals

Drug Profile

Sebelipase alfa - Alexion Pharmaceuticals

Alternative Names: Kanuma; Lysosomal acid lipase - Synageva BioPharma; Recombinant human lysosomal acid lipase - Synageva BioPharma; Recombinant lysosomal acid lipase - Synageva BioPharma; rhLAL - Synageva BioPharma; rLAL; SBC-102; Sebelipase alfa rce

Latest Information Update: 24 Mar 2018

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At a glance

  • Originator Synageva BioPharma
  • Developer Alexion Pharmaceuticals
  • Class Carboxylic ester hydrolases; Proteins
  • Mechanism of Action Sterol esterase replacements; Sterol esterase stimulants
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Wolman disease; Cholesterol ester storage disease
  • New Molecular Entity Yes

Highest Development Phases

  • Marketed Cholesterol ester storage disease; Wolman disease

Most Recent Events

  • 06 Feb 2018 Pooled efficacy and adverse events data from the phase II/III VITAL trial and the phase II CL08 trial in Wolman disease released by Alexion Pharmaceuticals
  • 01 Dec 2017 Alexion Pharmaceuticals completes a phase II trial in Cholesterol ester storage in US, Australia, Belgium, Brazil, Canada, Croatia, Denmark, Germany, Italy, Mexico, Netherlands, Russia, Spain, Turkey, UK (IV) (NCT02112994)
  • 20 Oct 2017 Efficacy and adverse events data from a phase II trial in Cholesterol ester storage disease presented at the 68th Annual Meeting of the American Association for the Study of Liver Diseases (AASLD-2017)
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