Product Description
Luspatercept, a recombinant fusion protein that binds transforming growth factor beta superfamily ligands to reduce SMAD2 and SMAD3 signaling, showed promising results in a phase 2 study. Luspatercept reduced the severity of anemia in patients with lower-risk myelodysplastic syndromes with ring sideroblasts who had been receiving regular red-cell transfusions and who had disease that was refractory to or unlikely to respond to erythropoiesis-stimulating agents or who had discontinued such agents owing to an adverse event. (Sourced from: https://www.nejm.org/doi/full/10.1056/nejmoa1908892)
Mechanisms of Action: TGFb Inhibitor
Novel Mechanism: No
Modality: Fusion Protein
Route of Administration: Subcutaneous
FDA Designation: Priority Review - Anemia|Myelodysplastic Syndrome *
Approval Status: Approved
Approved Countries: Australia | Austria | Belgium | Brazil | Canada | Chile | Croatia | Cyprus | Czech | Denmark | Egypt | Estonia | European Medicines Agency | Finland | France | Germany | Greece | Hong Kong | Hungary | Iceland | Ireland | Israel | Italy | Korea | Latvia | Lithuania | Luxembourg | Malaysia | Netherlands | Norway | Peru | Poland | Portugal | Romania | Russia | Saudi Arabia | Singapore | Slovakia | Slovenia | Spain | Sweden | Taiwan | Turkey | United Arab Emirates | United States | Uruguay
Approved Indications: None
Known Adverse Events: None
Company: Bristol-Myers Squibb
Company Location: NEW YORK NY 10016
Company CEO: Giovanni Caforio
Additonal Commercial Interests: None
Clinical Description
Countries in Clinic: Argentina, Australia, Austria, Belgium, Brazil, Bulgaria, Canada, Chile, China, Colombia, Czech Republic, France, Germany, Greece, Hong Kong, Hungary, India, Ireland, Israel, Italy, Japan, Korea, Lebanon, Lithuania, Malaysia, Mexico, Netherlands, Poland, Portugal, Puerto Rico, Romania, Russia, Saudi Arabia, Singapore, South Korea, Spain, Sweden, Switzerland, Taiwan, Thailand, Tunisia, Turkey, Ukraine, United Kingdom, United States
Active Clinical Trial Count: 47
Highest Development Phases
Phase 3: Anemia|Myelodysplastic Syndrome|Myelofibrosis|Myeloproliferative Disorders|Polycythemia|Polycythemia Vera|Preleukemia|Thrombocythemia, Essential|beta-Thalassemia
Phase 2: Anemia, Dyserythropoietic, Congenital|Anemia, Refractory|Anemia, Sideroblastic|Leukopenia|Neutropenia|Thalassemia|Thrombocytopenia|Thrombocytosis|alpha-Thalassemia
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
|---|---|---|---|---|---|
| ACE-536-B-THAL-004 | P2 |
Unknown Status |
beta-Thalassemia |
2034-02-26 |
|
| Phase 2 Study of Luspatercept in Adults with Alpha (α)-thalassemia | P2 |
Active, not recruiting |
Thalassemia |
2030-09-28 |
|
| CA056-025 | P3 |
Unknown Status |
Myelodysplastic Syndrome |
2030-03-11 |
|
| CASE2920 | P2 |
Recruiting |
Myelodysplastic Syndrome |
2029-08-01 |