This is a list of investigational autism and pervasive developmental disorder drugs, or drugs that are currently under development for clinical use in the treatment of autistic spectrum disorders and/or other pervasive developmental disorders but are not yet approved.
Chemical/generic names are listed first, with developmental code names, synonyms, and brand names in parentheses.
This list was last comprehensively updated in October 2024. It is likely to become outdated with time.
Under development
Preregistration
CM-4612 (CM-ADHD; CM-AT; CM-PK) – enzyme modulator/replacement for gastrointestinal or pancreatic secretory deficiencies[1]
Phase 3
Brexpiprazole (Rexulti) – dopamine D2 and D3 receptor partial agonist, serotonin 5-HT1A receptor partial agonist, serotonin 5-HT2A 5-HT2B, and 5-HT7 receptor antagonist, α1- and α2-adrenergic receptor antagonist, and atypical antipsychotic[2]
Bumetanideoralliquid (S-95008) – sodium–potassium–chloride symporter/cotransporter inhibitor and indirect GABAergic inhibitor[3][4]
Research programme: G protein-coupled receptor modulating small molecules - Omeros Corporation – G protein-coupled receptor modulator and neuromedin U receptor modulator[68]
Research programme: GPCR modulators - Nxera Pharma – various actions[69]
^Kaur H, Karabulut S, Gauld JW, Fagot SA, Holloway KN, Shaw HE, Fantegrossi WE (2023). "Balancing Therapeutic Efficacy and Safety of MDMA and Novel MDXX Analogues as Novel Treatments for Autism Spectrum Disorder". Psychedelic Medicine. 1 (3): 166–185. doi:10.1089/psymed.2023.0023. Author Disclosure Statement: H.K. is Vice President of Research at PharmAla Biotech and is listed as an inventor on patents related to the research in this review article. H.E.S. receives salary supported from a contract between PharmAla Biotech and UAMS. W.E.F. receives research funds and salary support from a contract between PharmAla Biotech and UAMS. The other authors have no conflicts to disclose.
^Lesko, Lori (13 November 2018). "Biomarkers for autism". Drug Discovery News. Retrieved 10 November 2024. According to Derham, "This drug (QBM-001), acts as an allosteric regulator of faulty channels in the brain to potentially alleviate the condition and allow toddlers to actively develop language and speech and avoid life-long speech and intellectual disability of being non-verbal. QBM-001 also reduces inflammation in the brain, and by so doing, can reduce the amount of long-term nerve loss."
^Siegel M, Beaulieu AA (August 2012). "Psychotropic medications in children with autism spectrum disorders: a systematic review and synthesis for evidence-based practice". J Autism Dev Disord. 42 (8): 1592–1605. doi:10.1007/s10803-011-1399-2. PMID22068820.
^Kisely S, Connor M, Somogyi AA, Siskind D (March 2023). "A systematic literature review and meta-analysis of the effect of psilocybin and methylenedioxymethamphetamine on mental, behavioural or developmental disorders". Aust N Z J Psychiatry. 57 (3): 362–378. doi:10.1177/00048674221083868. PMID35285280.
^Aman MG, Kern RA (July 1989). "Review of fenfluramine in the treatment of the developmental disabilities". J Am Acad Child Adolesc Psychiatry. 28 (4): 549–565. doi:10.1097/00004583-198907000-00014. PMID2670881.
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