luspatercept
Trade name: Reblozyl
Approved
Nov 8, 2019
Luspatercept, sold under the brand name Reblozyl, is a medication used for the treatment of anemia in beta thalassemia and myelodysplastic syndromes. — Wikipedia
Clinical trial activity
40 trials · 23 clinical orgs · 2 marketing orgs
Earliest trial started Sep 1, 2011 (NCT01432717)
Timeline
2010s
- Jan 1, 2011
Earliest Phase 1 Sponsor(trial)
- Jan 1, 2013
Earliest Phase 2 Sponsor(trial)
- Mar 11, 2013
Orphan Drug Designation
- Jan 1, 2016
- Nov 8, 2019
Celgene — Earliest FDA Approval
- Nov 8, 2019
Celgene — Marketing Organization
- Nov 8, 2019
Celgene — NDA Organization
- Nov 8, 2019
Bristol-Myers Squibb — NDA Secondary Org
Indications
Studied for
- Anemia · Phase 4
- Anemia, Aplastic · Phase 4
- beta-Thalassemia · Phase 4
- Blood Transfusion · Phase 4
- Chromosomes, Human, Pair 5 · Phase 2
- Clonal Hematopoiesis · Phase 2
- Erythrocyte Transfusion · Phase 3
- Fibrosis · Phase 2
- Leukopenia · Phase 2
- Myelodysplastic Syndromes · Phase 4
- Myeloproliferative Disorders · Phase 3
- Neutropenia · Phase 2
- Polycythemia Vera · Phase 3
- Primary Myelofibrosis · Phase 3
- Risk Factors · Phase 2
- Thalassemia · Phase 2
- Thrombocytopenia · Phase 2
Mechanism of action
- Activin receptor type-2BANTAGONIST
ACVR2B antagonist
- Transforming growth factor beta-1INHIBITOR
TGFB1 inhibitor
- Transforming growth factor betaINHIBITOR
Transforming growth factor beta inhibitor
Erythroid maturation agent. Luspatercept is a recombinant protein that mimics the human activin receptor type IIB and sequesters its TGF-beta ligands. This reduces the interaction between TGF-beta ligands and the native TGF-beta receptor reducing downstream Smad2/3 signaling with the overall result of increased erythroid maturation.
Approval history
- approvedPriority reviewFast trackNov 8, 2019
Chemistry & pharmacology
- Chirality
- Single Stereoisomer
- Inorganic
- No
- Polymer
- No
- Delivery
- Parenteral
- Availability
- Prescription Only
- Multi-specific
- No
Oral
No
Parenteral
Yes
Topical
No
Sources
- WikipediaLuspatercept ↗
- NCATSAQK7UBA1LS ↗
- ChEMBLCHEMBL3039545 ↗
Also known as
- ace-536
- ace-536
- ace-536
- luspatercept
- luspatercept
- luspatercept
Clinical trials
| NCT ID | Phase | Start date | Sponsor(s) |
|---|---|---|---|
| NCT07096297 | Phase 2 | Nov 30, 2025 | Bristol-Myers Squibb, Yale University |
| NCT06964971 | N/A | May 20, 2025 | Zhejiang Chinese Medical University |
| NCT06927232 | Phase 3 | Apr 30, 2025 | Chinese Academy of Medical Sciences |
| NCT06788691 | Phase 2 | Feb 28, 2025 | Bristol-Myers Squibb, Cornell University |
| NCT06517875 | Phase 2 | Dec 17, 2024 | GlaxoSmithKline |
| NCT06424639 | Phase 4 | May 31, 2024 | Chinese Academy of Medical Sciences |
| NCT06113302 | Early Phase 1 (Phase 1) | Apr 30, 2024 | University of Texas at Houston |
| NCT06164821 | N/A | Jan 1, 2024 | People's Liberation Army of China |
| NCT05949684 | Phase 3 | Oct 24, 2023 | Bristol-Myers Squibb |
| NCT06045689 | Phase 3 | Oct 5, 2023 | Bristol-Myers Squibb |
| NCT06006949 | Phase 4 | Aug 31, 2023 | Chinese Academy of Medical Sciences |
| NCT05925504 | Phase 2 | Jul 1, 2023 | Beijing Health Alliance Charitable Foundation, China Academy of Chinese Medical Sciences |
| NCT05891249 | Phase 4 | Jun 5, 2023 | Bristol-Myers Squibb |
| NCT05732961 | Phase 2 | Feb 21, 2023 | Bristol-Myers Squibb, University of South Florida |
| NCT05664737 | Phase 2 | Dec 9, 2022 | Bristol-Myers Squibb |
| NCT05924100 | Phase 2 | Nov 3, 2022 | Associazione Qol-one |
| NCT05567458 | Phase 2 | Oct 15, 2022 | Bristol-Myers Squibb |
| NCT05384691 | Phase 2 | Sep 27, 2022 | Celgene, University of Leipzig |
| NCT05462548 | Phase 4 | Jul 31, 2022 | Sun Yat-Sen University |
| NCT05399732 | Phase 2 | Jul 31, 2022 | Chinese Academy of Medical Sciences |
Organizations
Research & Development (23)
| Organization | Org type | Trials | As lead sponsor | Phases | Earliest year |
|---|---|---|---|---|---|
| Celgene | For profit | 14 | 10 | 2 | 2016 |
| Acceleron Pharma, Inc. | For profit | 10 | 5 | 3 | 2011 |
| Bristol-Myers Squibb | For profit | 10 | 5 | 3 | 2021 |
| Chinese Academy of Medical Sciences | Academic/Hospital | 4 | 4 | 3 | 2022 |
| Associazione Qol-one | Academic/Hospital | 1 | 1 | 1 | 2022 |
| Beijing Health Alliance Charitable Foundation | Academic/Hospital | 1 | 0 | 1 | 2023 |
| China Academy of Chinese Medical Sciences | Academic/Hospital | 1 | 1 | 1 | 2023 |
| Cornell University | Academic/Hospital | 1 | 1 | 1 | 2025 |
| GlaxoSmithKline | For profit | 1 | 1 | 1 | 2024 |
| Groupe Francophone des Myelodysplasies | Academic/Hospital | 1 | 1 | 1 | 2022 |
| GWT-TUD GmbH | For profit | 1 | 1 | 1 | 2021 |
| Hôpital Saint Louis | Academic/Hospital | 1 | 0 | 1 | 2022 |
| Mayo Clinic | Academic/Hospital | 1 | 1 | 1 | 2021 |
| National Cancer Institute (NCI) | Government | 1 | 0 | 1 | 2021 |
| People's Liberation Army of China | Government | 1 | 1 | 1 | 2024 |
| Sun Yat-Sen University | Academic/Hospital | 1 | 1 | 1 | 2022 |
| University of Leipzig | Academic/Hospital | 1 | 1 | 1 | 2022 |
| University of Miami | Academic/Hospital | 1 | 1 | 1 | 2021 |
| University of South Florida | Academic/Hospital | 1 | 1 | 1 | 2023 |
| University of Texas at Houston | Academic/Hospital | 1 | 1 | 1 | 2024 |
Marketing (3)
| Organization | Org type | Relationship | Date |
|---|---|---|---|
| Bristol-Myers Squibb | For profit | NDA2 | Nov 8, 2019 |
| Celgene | For profit | MKTG | Nov 8, 2019 |
| Celgene | For profit | NDA | Nov 8, 2019 |