Phuturdex › Diseases › Lysosomal storage disease
Drugs in development · updated 30 October 2026
Lysosomal storage disease: drugs in development
As of 30 October 2026 there are 12 active clinical trials (recruiting or ongoing, phases I–III) on 11 drugs for lysosomal storage disease. 11 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 |
|---|---|---|---|
| GNR-055 | II–III | 1 | NCT05208281 |
| OTL-200 | III | 1 | NCT04283227 |
| RGX-121 | II–III | 1 | NCT03566043 |
| 4D-310 | I–II | 2 | NCT05629559 |
| AB-1009 | I–II | 1 | NCT07282847 |
| DFT383 | I–II | 1 | NCT06910813 |
| FBX-101 | I–II | 1 | NCT05739643 |
| PBGM01 | I–II | 1 | NCT04713475 |
| PLX-200 | II | 1 | NCT07740512 |
| RP-A501 | II | 1 | NCT06092034 |
| SPK-3006 | I–II | 1 | NCT04093349 |
Sorted by most advanced phase and number of active trials.
All 11 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
- Mucopolysaccharidosis II 15 drugs
- Fabry disease 10 drugs
- Mucopolysaccharidosis III 8 drugs
- Glycogen storage disease II 9 drugs
- Mucopolysaccharidosis IV 3 drugs
- Niemann–Pick disease, type C 3 drugs
- Bone disease 9 drugs
- Musculoskeletal disorder 22 drugs
- Osteochondrodysplasia 3 drugs
- Mucopolysaccharidosis I 7 drugs
- Mucopolysaccharidosis VI 3 drugs
Broader categories
- Nutritional and metabolic diseases 5 drugs
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities 4 drugs
- Metabolism, Inborn Errors 6 drugs
- Metabolic disease 30 drugs
- Genetic disease 33 drugs
- Brain Diseases 23 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: D016464 “Lysosomal Storage Diseases”.