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Drugs in development · updated 30 October 2026

Fragile X syndrome: drugs in development

Rare diseasePrevalence 1-5 / 10 000 according to Orphanet · ORPHA:908

As of 30 October 2026 there are 8 active clinical trials (recruiting or ongoing, phases I–III) on 10 drugs for fragile X syndrome. 5 are experimental, that is not yet approved in the United States.

8Active trials
5Experimental 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
CodabakalnerII–III1NCT07439510
ZatolmilastBPN14770III1NCT05367960
ZYN002II–III1NCT03802799
CTH120II1NCT07654114
SRX246II1NCT07328529

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
BaclofenFleqsuvy®, Lyvispah®II1NCT05418049
MemantineNamenda, Memantine Mylan®II1NCT05418049
RoflumilastZoryve®, Daliresp®II1NCT05418049
SertralineZoloft®II1NCT06081348
MethylphenidateConcerta, Relexxii®I1NCT05301361

Sorted by most advanced phase and number of active trials.

All 10 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: D005600 “Fragile X Syndrome”. Prevalence: Orphanet.

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