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Drugs in development · updated 30 October 2026

Multiple system atrophy: drugs in development

Rare diseasePrevalence 1-9 / 100 000 according to Orphanet · ORPHA:102

As of 30 October 2026 there are 21 active clinical trials (recruiting or ongoing, phases I–III) on 19 drugs for multiple system atrophy. 15 are experimental, that is not yet approved in the United States.

21Active trials
15Experimental drugs
4Experimental, in phase III

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.

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Experimental drugs

Not yet approved by the US FDA for any indication.

DrugMost advanced phaseActive trialsReference trial
AmlenetugLu AF82422III2NCT06706622
ONO-2808III2NCT07825402
Butylphthalide3-N-butylphthalideII–III1NCT07518810
MSA-01III1NCT07446894
TEV-56286II2NCT07197866
ATH434II1NCT07729852
Foralumab NasalII1NCT06868628
TributyrinGlycerol TributyrateI–II1NCT07863180
YA-101II1NCT06848231
HOMSC300I1NCT05698017
ION464I1NCT04165486
SST001I1NCT07604116
ALT001Early I2NCT07476352
AleetoEarly I1NCT06765733
C11-SY08Early I1NCT06098612

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.

DrugMost advanced phaseActive trialsReference trial
18F-DOPA18F-FDOPA, Fluorodopa F 18I1NCT04246437
CarbidopaLodosyn®I1NCT04246437
EntacaponeComtan®, Entacapone Orion®I1NCT04246437
TrimethaphanArfonad®I1NCT02726711

Sorted by most advanced phase and number of active trials.

All 19 drugs, with the details

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Broader categories

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: D019578 “Multiple System Atrophy”. Prevalence: Orphanet.

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