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Drugs in development · updated 30 October 2026

Autism spectrum disorder: drugs in development

As of 30 October 2026 there are 41 active clinical trials (recruiting or ongoing, phases I–III) on 35 drugs for autism spectrum disorder. 14 are experimental, that is not yet approved in the United States.

41Active trials
14Experimental 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
ArbaclofenIII1NCT07655115
CodabakalnerII–III1NCT07439510
NTI164III1NCT07257939
CannabidivarinII1NCT03202303
DT402II1NCT07303907
KZ101II1NCT06866275
ML-004II1NCT05889273
SulforaphaneII1NCT02677051
TB006II1NCT06500637
TTYP01II1NCT06757504
ExosomesI2NCT07304440
GlutathioneI2NCT07304440
PsilocybinPsilocybineI1NCT06731621
VGN-Ex05eEarly I1NCT07433517

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
N-AcetylcysteineAcetylcysteine, AcetadoteII–III3NCT05494398
LumateperoneCaplyta®III1NCT06229210
LeucovorinFolinic acid, l-leucovorinII3NCT07642661
CannabidiolEpidiolex, Epidyolex®II2NCT04517799
LevoleucovorinLevofolinate, Levofolinic AcidII2NCT04060017
Oxytocin nasal sprayOxytocin 10 Usp Units In Dextrose 5%®, Oxytocin 5 Usp Units In Dextrose 5%®II2NCT05754073
All-trans retinoic acidATRA, TretinoinII1NCT07079696
DiphenhydramineBenadryl, Benadryl Preservative Free®II1NCT05501678
EscitalopramLexaproII1NCT06826326
EsomeprazoleNexium 24Hr®, Nexium®II1NCT03866668
GanaxoloneZtalmy®II1NCT07635862
MoviPrepII1NCT03408886
PimavanserinNuplazid®II1NCT05999240
PitolisantWakix®, Ozawade®II1NCT05953389
PropranololHemangeol®, Innopran Xl®II1NCT07091279

Sorted by most advanced phase and number of active trials. Showing 15 of 21 drugs.

All 35 drugs, with the details

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Specific forms

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: D000067877 “Autism Spectrum Disorder”.

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