Phuturdex › Diseases › Metabolism, Inborn Errors
Drugs in development · updated 30 October 2026
Metabolism, Inborn Errors: drugs in development
As of 30 October 2026 there are 7 active clinical trials (recruiting or ongoing, phases I–III) on 6 drugs for metabolism, Inborn Errors. 6 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 |
|---|---|---|---|
| ECUR-506 | III | 1 | NCT06255782 |
| Zodasiran | III | 1 | NCT07473843 |
| MRNA-3927 | I–II | 2 | NCT05130437 |
| FBX-101 | I–II | 1 | NCT05739643 |
| MRNA-3705 | I–II | 1 | NCT05295433 |
| CD19 | Early I | 1 | NCT02356653 |
Sorted by most advanced phase and number of active trials.
All 6 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
- Gaucher's disease 18 drugs
- Phenylketonuria 16 drugs
- Hyperlipoproteinemia Type II 12 drugs
- Mucopolysaccharidosis II 15 drugs
- Gout 13 drugs
- Fabry disease 10 drugs
- Congenital adrenal hyperplasia 7 drugs
- Lysosomal storage disease 11 drugs
- Glycogen storage disease II 9 drugs
- Hyperuricemia 13 drugs
- Mucopolysaccharidosis III 8 drugs
- Homozygous familial hypercholesterolemia 7 drugs
Broader categories
- Nutritional and metabolic diseases 5 drugs
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities 4 drugs
- Metabolic disease 30 drugs
- Neoplasm 671 drugs
- Digestive System Diseases 36 drugs
- Bone marrow disease 14 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: D008661 “Metabolism, Inborn Errors”.