lumasiran
Trade name: Oxlumo
Nucleic acidapprovedOrphan Drug FDABreakthrough Therapy FDA
Approved
Nov 23, 2020
Clinical trial activity
8 trials · 2 clinical orgs · 1 marketing orgs
Phase 1
1
Phase 2
7
Phase 3
3
Phase 4
0
Earliest trial started Mar 1, 2016 (NCT02706886)
Timeline
2010s
- Jan 1, 2016
Alnylam Pharmaceuticals — Earliest Phase 1 Sponsor(trial)
- Feb 8, 2016
Orphan Drug Designation
- Jan 1, 2017
Alnylam Pharmaceuticals — Earliest Phase 2 Sponsor(trial)
- Jan 1, 2018
Alnylam Pharmaceuticals — Earliest Phase 3 Sponsor(trial)
2020s
- Nov 23, 2020
Alnylam Pharmaceuticals — Earliest FDA Approval
- Nov 23, 2020
Alnylam Pharmaceuticals — Marketing Organization
- Nov 23, 2020
Alnylam Pharmaceuticals — NDA Secondary Org
- Nov 23, 2020
Alnylam Pharmaceuticals — NDA Organization
Indications
Studied for
- Angiotensinogen · Phase 2
- Cardiovascular Diseases · Phase 2
- Chronic Kidney Disease-Mineral and Bone Disorder · Phase 2
- Dialysis · Phase 2
- Heart Disease Risk Factors · Phase 2
- Hyperoxaluria · Phase 3
- Hyperoxaluria, Primary · Phase 3
- Kidney Diseases · Phase 2
- Oxalates · Phase 2
- RNAi Therapeutics · Phase 2
- RNA, Small Interfering · Phase 2
- Urination Disorders · Phase 2
Mechanism of action
- Hydroxyacid oxidase 1ANTAGONIST
HAO1 antagonist
- Hydroxyacid oxidase 1 mRNARNAI INHIBITOR
Hydroxyacid oxidase 1 mRNA RNAi inhibitor
Approval history
- approvedPriority reviewBreakthroughNov 23, 2020
Chemistry & pharmacology
- Chirality
- Single Stereoisomer
- Inorganic
- No
- Polymer
- No
- Availability
- Prescription Only
- Multi-specific
- No
Oral
No
Parenteral
No
Topical
No
First in class
Sources
- ChEMBLCHEMBL4650275 ↗
- ChEMBLCHEMBL4297779 ↗
Also known as
- aln-go1
Clinical trials
| NCT ID | Phase | Start date | Sponsor(s) |
|---|---|---|---|
| NCT06225544 | Phase 2 | Apr 14, 2024 | Alnylam Pharmaceuticals, Humboldt University |
| NCT05161936 | Phase 2 | Dec 31, 2021 | Alnylam Pharmaceuticals |
| NCT04152200 | Phase 3 | Nov 30, 2019 | Alnylam Pharmaceuticals |
| NCT03905694 | Phase 3 | Apr 22, 2019 | Alnylam Pharmaceuticals |
| NCT03681184 | Phase 3 | Sep 26, 2018 | Alnylam Pharmaceuticals |
| NCT03350451 | Phase 2 | Nov 20, 2017 | Alnylam Pharmaceuticals |
| NCT02706886 | Phase 1/Phase 2 (Phase 2) | Mar 1, 2016 | Alnylam Pharmaceuticals |
| NCT04125472 | N/A | — | Alnylam Pharmaceuticals |
Showing 8 of 8 trials
Page 1 / 1
Organizations
Research & Development (2)
| Organization | Org type | Trials | As lead sponsor | Phases | Earliest year |
|---|---|---|---|---|---|
| Alnylam Pharmaceuticals | For profit | 8 | 7 | 3 | 2016 |
| Humboldt University | Academic/Hospital | 1 | 1 | 1 | 2024 |
2 organizations
Page 1 / 1
Marketing (3)
| Organization | Org type | Relationship | Date |
|---|---|---|---|
| Alnylam Pharmaceuticals | For profit | MKTG | Nov 23, 2020 |
| Alnylam Pharmaceuticals | For profit | NDA2 | Nov 23, 2020 |
| Alnylam Pharmaceuticals | For profit | NDA | Nov 23, 2020 |