Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
D0CH4A
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| Former ID |
DIB010835
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| Drug Name |
SBC-102
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| Synonyms |
Sebelipase alfa; Enzyme replacement therapy (protein recombinant, lysosomal acid lipase deficiency), Synageva BioPharma
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| Indication | Enzyme deficiency [ICD-11: 5C51-5C57] | Approved | [1] | |
| Company |
Synageva biopharma
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| ADReCS Drug ID | BADD_D01996 | |||
| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Lysosomal acid lipase (LIPA) | Target Info | Modulator | [1], [2] |
| KEGG Pathway | Steroid biosynthesis | |||
| Lysosome | ||||
| Panther Pathway | Androgen/estrogene/progesterone biosynthesis | |||
| Pathwhiz Pathway | Steroid Biosynthesis | |||
| Bile Acid Biosynthesis | ||||
| WikiPathways | Phase I biotransformations, non P450 | |||
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT01757184) A Multicenter Study of SBC-102 (Sebelipase Alfa) in Patients With Lysosomal Acid Lipase Deficiency/ ARISE (Acid Lipase Replacement Investigating Safety and Efficacy).U.S. National Institutes of Health. | |||
| REF 2 | A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency. N Engl J Med. 2015 Sep 10;373(11):1010-20. | |||
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