Phuturdex › Diseases › Aplastic anemia
Drugs in development · updated 30 October 2026
Aplastic anemia: drugs in development
As of 30 October 2026 there are 41 active clinical trials (recruiting or ongoing, phases I–III) on 43 drugs for aplastic anemia. 9 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 |
|---|---|---|---|
| CD34 | I–II | 1 | NCT01966367 |
| CK0801 | II | 1 | NCT07499102 |
| CM313 injection | I–II | 1 | NCT06791824 |
| Cyclosporins | II | 1 | NCT01659606 |
| DibotatugDR-01 | I–II | 1 | NCT07712562 |
| Golidocitinib | I–II | 1 | NCT07297550 |
| Recombinant human thrombopoietin | II | 1 | NCT07400341 |
| RimiducidAP1903 | I–II | 1 | NCT03301168 |
| RD13-02 | I | 1 | NCT06622694 |
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 |
|---|---|---|---|
| EltrombopagRevolade, Eltrombopag Accord® | III | 3 | NCT06493981 |
| AvatrombopagDoptelet Sprinkle®, Doptelet® | II–III | 1 | NCT07786922 |
| N-AcetylcysteineAcetylcysteine, Acetadote | III | 1 | NCT07737262 |
| FludarabineFludara, FLUDARABIN | I–II | 11 | NCT03622788 |
| CyclophosphamideCyclophosphamid, Cytoxan | I–II | 10 | NCT03622788 |
| CyclosporineCyclosporine A, Cyclosporin Capsules | II | 6 | NCT01624805 |
| Mycophenolate MofetilMycophenolate, Cellcept | I–II | 6 | NCT03192397 |
| SirolimusRapamycin, Scomara® | I–II | 6 | NCT07796620 |
| MelphalanEvomela®, Hepzato® | I–II | 5 | NCT03192397 |
| Anti-Thymocyte Globulin | I–II | 4 | NCT03622788 |
| RuxolitinibJakavi, Jakafi Xr® | I–II | 4 | NCT05998408 |
| TacrolimusEnvarsus XR, Prograf® | II | 4 | NCT06412497 |
| RituximabRuxience, Rituxan | II | 3 | NCT06412497 |
| AlemtuzumabCampath, Lemtrada® | II | 2 | NCT01659606 |
| BusulfanBusulphan, Busulfex® | II | 2 | NCT00544115 |
Sorted by most advanced phase and number of active trials. Showing 15 of 34 drugs.
All 43 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 →Specific forms
- Fanconi anemia 16 drugs
- Diamond-Blackfan anemia 8 drugs
- Primary immunodeficiency disease 13 drugs
- Dyskeratosis congenita 15 drugs
- Beta thalassemia 34 drugs
- Sickle-cell disease 73 drugs
- Immune Deficiency Disease 6 drugs
- Bone marrow failure 26 drugs
- Hoyeraal-Hreidarsson syndrome 9 drugs
- Myelodysplastic Syndromes 203 drugs
- Acute myeloid leukemia 314 drugs
- Cytopenia 14 drugs
Broader categories
- Anemia 52 drugs
- Bone marrow disease 14 drugs
- Hematologic Diseases 57 drugs
- Bone marrow failure 26 drugs
- Immune System Diseases 54 drugs
- Lymphoproliferative disorders 51 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: D000741 “Anemia, Aplastic”.