| Pharmaceutical Information |
| Drug Name |
Sebelipase alfa |
| Drug ID |
BADD_D01996 |
| Description |
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.[L39337]
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).[L39337,L39342] |
| Indications and Usage |
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.[L39337,L39342] |
| Marketing Status |
approved; investigational |
| ATC Code |
A16AB14 |
| DrugBank ID |
DB11563
|
| KEGG ID |
D10377
|
| MeSH ID |
C000603932
|
| PubChem ID |
Not Available
|
| TTD Drug ID |
D0CH4A
|
| NDC Product Code |
63508-003; 69443-400; 25682-007 |
| UNII |
K4YTU42T8G
|
| Synonyms |
Sebelipase alfa | Kanuma |
|
| Chemical Information |
| Molecular Formula |
Not Available |
| CAS Registry Number |
1276027-63-4 |
| SMILES |
Not Available |
| Chemical Structure |
|
|
| ADRs Induced by Drug |
|
|
*The priority for ADR severity classification is based on FAERS assessment, followed by the most severe level in CTCAE rating. If neither is available, it will be displayed as 'Not available'.
**The 'Not Available' level is hidden by default and can be restored by clicking on the legend twice..
|
|
|
