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Sebelipase alfa - Alexion AstraZeneca Rare Disease

Drug Profile

Sebelipase alfa - Alexion AstraZeneca Rare Disease

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

Latest Information Update: 05 Nov 2023

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At a glance

  • Originator Synageva BioPharma
  • Developer Alexion AstraZeneca Rare Disease
  • Class Carboxylic ester hydrolases; Proteins; Recombinant proteins
  • Mechanism of Action Sterol esterase replacements
  • 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

  • 27 Sep 2022 No development reported - Phase-II/III for Wolman disease (In infants, In neonates) in Ireland, United Kingdom, Italy, USA, Germany, Egypt, France (IV)
  • 21 Jul 2021 Alexion Pharmaceuticals has been acquired by AstraZeneca and changed its name to Alexion AstraZeneca Rare Disease
  • 13 Nov 2020 Pooled efficacy and adverse events data from a phase I LAL-CL01, phase II LAL-CL04 and LAL-CL06 and a phase III LAL-CL02 trial in Wolman disease and Cholesterol ester storage disease presented at the 70th Annual Meeting of the American Association for the Study of Liver Diseases (AASLD-2020)
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