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 rceLatest Information Update: 05 Nov 2023
At a glance
- Originator Synageva BioPharma
- Developer Alexion AstraZeneca Rare Disease
- Class Carboxylic ester hydrolases; Proteins; Recombinant proteins
- Mechanism of Action Sterol esterase replacements
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- 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)