Phuturdex › Diseases › Beta thalassemia
Drugs in development · updated 30 October 2026
Beta thalassemia: drugs in development
As of 30 October 2026 there are 26 active clinical trials (recruiting or ongoing, phases I–III) on 34 drugs for beta thalassemia. 11 are experimental, that is not yet approved in the United States.
Summary updated every week · data as of 30 October 2026. In Phuturdex you get the full detail of every drug and an email when an alert is published on the ones you follow.
Sign in free →Experimental drugs
Not yet approved by the US FDA for any indication.
| Drug | Most advanced phase | Active trials | Reference trial |
|---|---|---|---|
| Alpha Lipoic Acid | III | 1 | NCT07157722 |
| ALS20 | I–II | 1 | NCT06364774 |
| BriquilimabJSP191 | I–II | 1 | NCT05357482 |
| BRL-101 | I–II | 1 | NCT05577312 |
| EDIT-301 | I–II | 1 | NCT05444894 |
| HGI-001 | II | 1 | NCT07721480 |
| REGN7999 | II | 1 | NCT06421636 |
| 9MW3011 | I | 1 | NCT06772766 |
| BCL11A | I | 1 | NCT06647979 |
| CS-101 | Early I | 1 | NCT06328764 |
| GMCN-508B | Early I | 1 | NCT05762510 |
Sorted by most advanced phase and number of active trials.
Approved drugs in new trials
Approved by the US FDA for at least one indication, not necessarily this disease, and now in active trials on it.
| Drug | Most advanced phase | Active trials | Reference trial |
|---|---|---|---|
| CTX001 | III | 3 | NCT04208529 |
| LuspaterceptACE-536, Reblozyl | III | 3 | NCT04064060 |
| ThalidomideThalomid®, Thalidomide Lipomed® | III | 3 | NCT07884773 |
| DanazolDanocrine® | III | 1 | NCT07884773 |
| N-AcetylcysteineAcetylcysteine, Acetadote | III | 1 | NCT07157722 |
| PrednisolonePrednisolon, Prenisolone | III | 1 | NCT07884773 |
| AlemtuzumabCampath, Lemtrada® | I–II | 5 | NCT07599176 |
| HydroxyureaHydroxycarbamide, Hydroxycarbamid | I–II | 4 | NCT03653338 |
| FludarabineFludara, FLUDARABIN | I–II | 3 | NCT03653338 |
| RituximabRuxience, Rituxan | I–II | 2 | NCT03653338 |
| SirolimusRapamycin, Scomara® | I–II | 2 | NCT00061568 |
| ThiotepaTepylute®, Tepadina And Sodium Chloride® | I–II | 2 | NCT03653338 |
| AbataceptOrencia® | I–II | 1 | NCT07599176 |
| BortezomibVelcade, Bortezomib Hospira® | II | 1 | NCT05736419 |
| CyclophosphamideCyclophosphamid, Cytoxan | II | 1 | NCT05736419 |
Sorted by most advanced phase and number of active trials. Showing 15 of 23 drugs.
All 34 drugs, with the details
In Phuturdex every drug has all its trials, sponsors and countries, FDA and EMA approvals, orphan designations and safety alerts. Free until 31 March 2027.
Open in Phuturdex →Broader categories
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities 4 drugs
- Hemolytic anemia 6 drugs
- Anemia 52 drugs
- Congenital hemolytic anemia 4 drugs
- Hematologic Diseases 57 drugs
- Genetic disease 33 drugs
Source: Phuturdex analysis of ClinicalTrials.gov data (U.S. National Library of Medicine), updated 30 October 2026. Active trials = recruiting or ongoing, phases I–III, with at least one identified drug; phase and sites refer to the trials on this disease. Disease name: Wikidata; NLM MeSH term: D017086 “beta-Thalassemia”. Prevalence: Orphanet.