Sebelipase alfa

Drug Profile

Sebelipase alfa

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: 15 Nov 2017

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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

  • 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)
  • 20 Oct 2017 Long term safety and efficacy data from the phase III ARISE trial in Cholesterol ester storage disease presented at the Liver Meeting 2017: 70th Annual Meeting of the American Association for the Study of Liver Disease (AASLD-2017)
  • 18 Sep 2017 Registered for Cholesterol ester storage disease (In children, In adults, In adolescents) in Australia (IV)
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