Phuturdex › Diseases › Congenital Abnormalities
Drugs in development · updated 30 October 2026
Congenital Abnormalities: drugs in development
As of 30 October 2026 there are 8 active clinical trials (recruiting or ongoing, phases I–III) on 14 drugs for congenital Abnormalities. 3 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 |
|---|---|---|---|
| CTH120 | II | 1 | NCT07654114 |
| Miransertib | II | 1 | NCT04980872 |
| AIR-001 | I | 1 | NCT07431112 |
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 |
|---|---|---|---|
| TacrolimusEnvarsus XR, Prograf® | II–III | 2 | NCT02395497 |
| CyclophosphamideCyclophosphamid, Cytoxan | II | 2 | NCT02094794 |
| EtoposideAvopef®, Etopophos Preservative Free® | II | 2 | NCT02094794 |
| FludarabineFludara, FLUDARABIN | I–II | 2 | NCT00544466 |
| MelphalanEvomela®, Hepzato® | I–II | 2 | NCT00544466 |
| BusulfanBusulphan, Busulfex® | II | 1 | NCT00544115 |
| CyclosporineCyclosporine A, Cyclosporin Capsules | II | 1 | NCT00544115 |
| MethotrexateJylamvo®, Reditrex® | II | 1 | NCT00544115 |
| Mycophenolate MofetilMycophenolate, Cellcept | II | 1 | NCT00544115 |
| SirolimusRapamycin, Scomara® | II | 1 | NCT00544115 |
| Lactated ringers | I | 1 | NCT03723564 |
Sorted by most advanced phase and number of active trials.
All 14 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
- Autosomal dominant polycystic kidney 30 drugs
- Down syndrome 36 drugs
- Congenital adrenal hyperplasia 7 drugs
- Vascular malformation 9 drugs
- Tuberous sclerosis 10 drugs
- Hereditary hemorrhagic telangiectasia 8 drugs
- Lymphatic Abnormalities 7 drugs
- Epidermolysis bullosa dystrophica 8 drugs
- Angelman syndrome 4 drugs
- Fragile X syndrome 10 drugs
- Dyskeratosis congenita 15 drugs
- Congenital heart disease 8 drugs
Broader categories
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities 4 drugs
- Neoplasm 671 drugs
- Myeloid leukemia 26 drugs
- Hematologic Diseases 57 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: NLM MeSH; NLM MeSH term: D000013 “Congenital Abnormalities”.