Phuturdex › Diseases › Mucopolysaccharidoses
Drugs in development · updated 30 October 2026
Mucopolysaccharidoses: drugs in development
As of 30 October 2026 there are 1 active clinical trials (recruiting or ongoing, phases I–III) on 5 drugs for mucopolysaccharidoses. 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 |
| FludarabineFludara, FLUDARABIN | II | 1 | NCT01962415 |
| HydroxyureaHydroxycarbamide, Hydroxycarbamid | II | 1 | NCT01962415 |
| MelphalanEvomela®, Hepzato® | II | 1 | NCT01962415 |
| ThiotepaTepylute®, Tepadina And Sodium Chloride® | II | 1 | NCT01962415 |
Sorted by most advanced phase and number of active trials.
All 5 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
- Mucopolysaccharidosis II 15 drugs
- Mucopolysaccharidosis III 8 drugs
- Mucopolysaccharidosis IV 3 drugs
- Bone disease 9 drugs
- Musculoskeletal disorder 22 drugs
- Osteochondrodysplasia 3 drugs
- Genetic disease 33 drugs
- Mucopolysaccharidosis VI 3 drugs
- Dwarfism 3 drugs
- Mucopolysaccharidosis I 7 drugs
- Achondroplasia 8 drugs
- Lysosomal storage disease 11 drugs
Broader categories
- Digestive System Diseases 36 drugs
- Metabolic brain disease 3 drugs
- Brain Diseases 23 drugs
- Central nervous system disease 38 drugs
- Nervous System Diseases 39 drugs
- Metabolic disease 30 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: D009083 “Mucopolysaccharidoses”. Prevalence: Orphanet.