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Also known as Deferitrin (GT56-252)
Deferitrin · 1 trial · 1 indication
| Name | Type | Description |
|---|---|---|
| Deferitrin (GT56-252) | DRUG | - |
| desferoxamine (DFO) | DRUG | - |
Inclusion Criteria: * Beta-thalassemia patients, 18 years of age or older, currently undergoing chronic blood transfusion therapy and iron chelation therapy who weigh more than 40 kg. * No clinically significant findings on physical exam, medical history, or screening laboratories. * Serum ferritin...
Top 6 of 8 competitors
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| Agios Pharmaceuticals, Inc. | AGIO | 5 | PHASE3 | Mitapivat, AG-348 |
| Bristol-Myers Squibb Company | BMY | 6 | PHASE3 | Luspatercept |
| Vertex Pharmaceuticals Incorporated | VRTX | 3 | PHASE3 | CTX001 |
| Novo Nordisk A/S Sponsored ADR Class B | NVO | 2 | PHASE3 | Etavopivat |
| Regeneron Pharmaceuticals, Inc. | REGN | 1 | PHASE2 | REGN7999 |
| Editas Medicine, Inc. | EDIT | 2 | PHASE1 | EDIT-301 |
Deferitrin is an investigational small molecule being studied for beta-thalassemia, a blood disorder that requires regular transfusions and can lead to iron overload. It was evaluated in a Phase 1 study of patients with transfusional iron overload secondary to beta-thalassemia. Deferitrin has not been approved for any indication.
Deferitrin is being developed by Sanofi, which trades on the New York Stock Exchange under the ticker SNY. Sanofi sponsored the clinical research of Deferitrin in beta-thalassemia. No other developer or partner is identified for this asset.
Deferitrin is in Phase 1 clinical development. One Phase 1 trial has been completed, and no active trials are ongoing. The program has studied 25 patients in total. Deferitrin remains investigational and is not approved by the FDA for any use.
Deferitrin has been studied in one completed Phase 1 trial, NCT00069862, titled Iron Balance Study of DFO and GT56-252 in Patients With Transfusional Iron Overload Secondary to Beta-Thalassemia. The trial enrolled 25 patients in the United States, ages 18 and older, and was uncontrolled, not randomized, and not double-blind.
Yes, Deferitrin is also known as GT56-252. The two names refer to the same investigational small molecule developed by Sanofi for beta-thalassemia. The completed Phase 1 trial NCT00069862 used the GT56-252 designation in its title.