Phuturdex › Diseases › Congenital bone marrow failure syndromes
Drugs in development · updated 30 October 2026
Congenital bone marrow failure syndromes: drugs in development
As of 30 October 2026 there are 3 active clinical trials (recruiting or ongoing, phases I–III) on 8 drugs for congenital bone marrow failure syndromes. 0 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 →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 |
|---|---|---|---|
| AlemtuzumabCampath, Lemtrada® | II | 1 | NCT01962415 |
| DecitabineDacogen® | II | 1 | NCT05796570 |
| FilgrastimRecombinant Human Granulocyte Colony-Stimulating Factor, Neupogen | II | 1 | NCT05796570 |
| FludarabineFludara, FLUDARABIN | II | 1 | NCT01962415 |
| HydroxyureaHydroxycarbamide, Hydroxycarbamid | II | 1 | NCT01962415 |
| MelphalanEvomela®, Hepzato® | II | 1 | NCT01962415 |
| ThiotepaTepylute®, Tepadina And Sodium Chloride® | II | 1 | NCT01962415 |
| ImatinibSTI571, Imkeldi® | I | 1 | NCT06090669 |
Sorted by most advanced phase and number of active trials.
All 8 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
- Bone marrow failure 26 drugs
- Dyskeratosis congenita 15 drugs
- Aplastic anemia 43 drugs
- Hoyeraal-Hreidarsson syndrome 9 drugs
- Sickle-cell disease 73 drugs
- Diamond-Blackfan anemia 8 drugs
- Myelodysplastic Syndromes 203 drugs
- Primary immunodeficiency disease 13 drugs
- Revesz syndrome 7 drugs
- Immune Deficiency Disease 6 drugs
- Acute myeloid leukemia 314 drugs
Broader categories
- Bone marrow disease 14 drugs
- Hematologic Diseases 57 drugs
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities 4 drugs
- Bone marrow failure 26 drugs
- Neoplasm 671 drugs
- Digestive System Diseases 36 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: D000080984 “Congenital Bone Marrow Failure Syndromes”.