Phuturdex › Diseases › Inborn metabolic brain disease
Drugs in development · updated 30 October 2026
Inborn metabolic brain disease: drugs in development
As of 30 October 2026 there are 3 active clinical trials (recruiting or ongoing, phases I–III) on 3 drugs for inborn metabolic brain disease. 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 |
|---|---|---|---|
| ECUR-506 | III | 1 | NCT06255782 |
| FBX-101 | I–II | 1 | NCT05739643 |
| DUOC-01 | I | 1 | NCT02254863 |
Sorted by most advanced phase and number of active trials.
All 3 drugs, with the details
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Open in Phuturdex →Specific forms
- Gaucher's disease 18 drugs
- Phenylketonuria 16 drugs
- Fabry disease 10 drugs
- Glycogen storage disease II 9 drugs
- Hepatolenticular Degeneration 7 drugs
- Lysosomal storage disease 11 drugs
- Niemann–Pick disease, type C 3 drugs
- Ornithine carbamoyltransferase deficiency 6 drugs
- Rare disease 12 drugs
- Glycogen storage disease 4 drugs
- Mitochondrial disease 10 drugs
- MELAS syndrome 3 drugs
Broader categories
- Nutritional and metabolic diseases 5 drugs
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities 4 drugs
- Neurodegenerative Diseases 25 drugs
- Nervous system heredodegenerative disease 3 drugs
- Digestive System Diseases 36 drugs
- Metabolic brain disease 3 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: D020739 “Brain Diseases, Metabolic, Inborn”.