Sebelipase alfa
- Generic Name
- Sebelipase alfa
- Brand Names
- Kanuma
- Drug Type
- Biotech
- Chemical Formula
- -
- CAS Number
- 1276027-63-4
- Unique Ingredient Identifier
- K4YTU42T8G
- Background
The lysosomal acid lipase (LAL) enzyme is found in lysosomes and is primarily responsible for the metabolism of lipids, and its absence or deficiency results in the accumulation of lipids in various organs. This lipid accumulation can lead to end-organ damage in the form of liver dysfunction or malabsorption secondary to intestinal dysfunction. In addition, patients with LAL deficiency typically develop dyslipidemia, which itself contributes to a number of adverse cardiovascular outcomes.
Sebelipase alfa is a recombinant form LAL approved for the treatment of LAL deficiency. It was first approved by both the FDA and EMA in 2015 and is marketed under the brand name Kanuma (Alexion Pharmaceuticals).
- Indication
Sebelipase alfa is a hydrolytic lysosomal cholesteryl ester and triacylglycerol-specific enzyme indicated for the treatment of patients with a diagnosis of Lysosomal Acid Lipase (LAL) deficiency.
- Associated Conditions
- Lysosomal Acid Lipase Deficiency
- Associated Therapies
- -
An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase Deficiency
- Conditions
- First Posted Date
- 2015-03-03
- Last Posted Date
- 2016-06-08
- Lead Sponsor
- Alexion Pharmaceuticals, Inc.
- Registration Number
- NCT02376751
Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency
- First Posted Date
- 2014-07-18
- Last Posted Date
- 2019-11-18
- Lead Sponsor
- Alexion Pharmaceuticals, Inc.
- Target Recruit Count
- 10
- Registration Number
- NCT02193867
Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency
- First Posted Date
- 2014-04-14
- Last Posted Date
- 2019-12-04
- Lead Sponsor
- Alexion Pharmaceuticals, Inc.
- Target Recruit Count
- 31
- Registration Number
- NCT02112994
Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency
- Conditions
- Interventions
- First Posted Date
- 2012-12-28
- Last Posted Date
- 2020-12-29
- Lead Sponsor
- Alexion Pharmaceuticals, Inc.
- Target Recruit Count
- 66
- Registration Number
- NCT01757184
Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency
- Conditions
- Interventions
- First Posted Date
- 2011-12-08
- Last Posted Date
- 2018-07-20
- Lead Sponsor
- Alexion Pharmaceuticals, Inc.
- Target Recruit Count
- 8
- Registration Number
- NCT01488097
Drug Development Updates
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