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Drugs in development · updated 30 October 2026

Huntington's disease: drugs in development

Rare diseasePrevalence 1-9 / 100 000 according to Orphanet · ORPHA:399

As of 30 October 2026 there are 16 active clinical trials (recruiting or ongoing, phases I–III) on 14 drugs for Huntington's disease. 10 are experimental, that is not yet approved in the United States.

16Active trials
10Experimental drugs
3Experimental, 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
SKY-0515II–III3NCT07378644
VotoplamIII2NCT07326709
PridopidineNurzigma®III1NCT07609108
HNSC-01I–II1NCT07451613
SPK-10001I–II1NCT06826612
TominersenII1NCT05686551
VO659I–II1NCT05822908
ALN-HTT02I1NCT06585449
RG6496I1NCT07246941
SRP-1005I1NCT07536061

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
DeutetrabenazineAustedo Xr®, Austedo®II–III1NCT04713982
ValbenazineIngrezza Sprinkle®, Ingrezza®III1NCT06312189
BiotinII1NCT04478734
ThiamineII1NCT04478734

Sorted by most advanced phase and number of active trials.

All 14 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: D006816 “Huntington Disease”. Prevalence: Orphanet.

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