Phuturdex › Diseases › Bone marrow failure
Drugs in development · updated 30 October 2026
Bone marrow failure: drugs in development
As of 30 October 2026 there are 16 active clinical trials (recruiting or ongoing, phases I–III) on 26 drugs for bone marrow failure. 7 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 |
| Cyclosporins | II | 1 | NCT01659606 |
| DibotatugDR-01 | I–II | 1 | NCT07712562 |
| RP-L102 | II | 1 | NCT04248439 |
| EXG34217 | I | 1 | NCT04211714 |
| CD19 | Early I | 1 | NCT02356653 |
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 |
|---|---|---|---|
| AvatrombopagDoptelet Sprinkle®, Doptelet® | II–III | 1 | NCT07786922 |
| FludarabineFludara, FLUDARABIN | I–II | 6 | NCT03622788 |
| CyclophosphamideCyclophosphamid, Cytoxan | I–II | 4 | NCT03622788 |
| AlemtuzumabCampath, Lemtrada® | II | 3 | NCT01659606 |
| Mycophenolate MofetilMycophenolate, Cellcept | II | 3 | NCT01659606 |
| Anti-Thymocyte Globulin | I–II | 2 | NCT03622788 |
| BusulfanBusulphan, Busulfex® | II | 2 | NCT04644016 |
| MelphalanEvomela®, Hepzato® | II | 2 | NCT03579875 |
| TacrolimusEnvarsus XR, Prograf® | II | 2 | NCT01659606 |
| ClofarabineClolar®, Evoltra® | II | 1 | NCT04644016 |
| CyclosporineCyclosporine A, Cyclosporin Capsules | II | 1 | NCT04644016 |
| EmapalumabGamifant® | II | 1 | NCT06430788 |
| MesnaUromitexan, Mesnex® | II | 1 | NCT04558736 |
| MethylprednisoloneMethylprednisolon, Methylprenisolone | II | 1 | NCT03579875 |
| RituximabRuxience, Rituxan | II | 1 | NCT03579875 |
Sorted by most advanced phase and number of active trials. Showing 15 of 19 drugs.
All 26 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
- Aplastic anemia 43 drugs
- Myelodysplastic Syndromes 203 drugs
- Acute myeloid leukemia 314 drugs
- Fanconi anemia 16 drugs
- Non-Hodgkin lymphoma 269 drugs
- Graft-versus-host disease 49 drugs
- Hodgkin lymphoma 93 drugs
- Multiple myeloma 272 drugs
- Precursor lymphoblastic lymphoma/leukemia 163 drugs
- Leukemia, Myelogenous, Chronic, BCR-ABL Positive 68 drugs
- Myeloproliferative disorders 91 drugs
- Myelofibrosis 91 drugs
Broader categories
- Bone marrow disease 14 drugs
- Hematologic Diseases 57 drugs
- Anemia 52 drugs
- Genetic disease 33 drugs
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities 4 drugs
- Immune System Diseases 54 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: D000080983 “Bone Marrow Failure Disorders”.