Drugs / sebelipase alfa
sebelipase alfa
Regulatory milestones approvals, filings & regulatory actions · 3 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Label expansion | US (FDA) | KANUMA | — | 2021-11-19 | fda.gov ↗ |
| Approved | US (FDA) | KANUMA | — | 2015-12-08 | fda.gov ↗ |
| Approved | EU (EMA) | Kanuma | — | 2015-08-28 | europa.eu ↗ |
Trials 7
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT01307098 | Apr 2011 → Jan 2012 | cholesteryl ester storage disease | Alexion Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 23 trials | ||||||
| Phase 2 | NCT02112994 | Jun 2014 → Dec 2017 | lysosomal acid lipase deficiency | Alexion Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 2 | NCT02193867 | Jun 2014 → Oct 2018 | lysosomal acid lipase deficiency | Alexion Pharmaceuticals, Inc. | Terminated | No outcome recorded |
| Phase 2 | NCT01488097 | Dec 2011 → Jun 2017 | cholesteryl ester storage disease | Alexion Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 2/31 trial | ||||||
| Phase 2/3 | NCT01371825 | May 2011 → Jan 2018 | Wolman disease | Alexion Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 31 trial · 1 met primary | ||||||
| Phase 3 | NCT01757184 | Jan 2013 → May 2014 | lysosomal acid lipase deficiency | Alexion Pharmaceuticals, Inc. | Completed | Met primary |
| Phase not stated1 trial | ||||||
| — | NCT02376751 | — | lysosomal acid lipase deficiency | Alexion Pharmaceuticals, Inc. | No longer available | No outcome recorded |
Evidence & citations 7 cited values
Every value below carries the sentence it was read from. 9 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | sebelipase alfa | ClinicalTrials.gov intervention name — accepted as the source's own label NCT02376751 ↗ |
| Known as | Kanuma | “In preclinical studies and clinical studies in participants with LAL-D, treatment with SBC-102 (sebelipase alfa, Kanuma®) has been shown to produce improvements in markers of...” NCT01757184 ↗ |
| Known as | SBC-102 | “Sebelipase alfa (SBC-102)” NCT01371825 ↗ |
| Known as | Sebelipasa alfa | ChEMBL registry synonym — accepted as the source's own label CHEMBL3039537 ↗ |
| Action | Restore | “replacement of the defective LAL enzyme with sebelipase alfa” PMID 34774639 ↗ Nov 2021 |
| Modality | Protein / enzyme biologic | “Sebelipase alfa is a recombinant human lysosomal acid lipase.” NCT01307098 ↗ |
| Route | Intravenous | “Participants received intravenous (IV) infusions of sebelipase alfa” NCT01371825 ↗ |