List of investigational autism and pervasive developmental disorder drugs explained

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 It is likely to become outdated with time.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

Phase 3

Phase 2

Phase 1

Preclinical

Research

Phase unknown

Not under development

Development suspended

No development reported

Discontinued

Clinically used drugs

Approved drugs

Off-label drugs

See also

External links

Notes and References

  1. Web site: CM 4612 . AdisInsight .
  2. Web site: Brexpiprazole - Lundbeck/Otsuka . AdisInsight .
  3. Web site: Bumetanide oral - Neurochlore . AdisInsight .
  4. Web site: Cariprazine - Gedeon Richter . AdisInsight .
  5. Web site: Lumateperone - Intra-Cellular Therapies . AdisInsight .
  6. Web site: Lurasidone - Sumitomo Pharma . AdisInsight .
  7. Web site: Tasimelteon - Vanda Pharmaceuticals . AdisInsight .
  8. Web site: AB 2004 . AdisInsight .
  9. Web site: Acamprosate - Confluence Pharmaceuticals . AdisInsight .
  10. Web site: Alogabat - Genentech . AdisInsight .
  11. Web site: Arbaclofen - Seaside Therapeutics . AdisInsight .
  12. Web site: Cannabidiol - GW Pharmaceuticals . AdisInsight .
  13. Web site: Cannabidiol - Zynerba Pharmaceuticals . AdisInsight .
  14. Web site: Cannabidivarin - GW Pharmaceuticals . AdisInsight .
  15. Web site: CP 101 - Finch Therapeutics Group . AdisInsight .
  16. Web site: JNJ 42165279 . AdisInsight .
  17. Web site: Oxytocin - Tonix Pharmaceuticals . AdisInsight .
  18. Web site: Pitolisant - Bioprojet . AdisInsight .
  19. Web site: Racemetirosine - Yamo Pharmaceuticals . AdisInsight .
  20. Web site: Suramin - PaxMedica . AdisInsight .
  21. Web site: ML 004 . AdisInsight .
  22. Web site: R(-)-Methylenedioxymetamfetamine - Mindmed . AdisInsight .
  23. Web site: STP 1 . AdisInsight .
  24. Web site: Sulforafan alfadex - TheraCryf . AdisInsight .
  25. Web site: Bryostatin 1 - Synaptogenix . AdisInsight .
  26. Web site: CJRB 303 . AdisInsight .
  27. Web site: Debamestrocel - BrainStorm Cell Therapeutics . AdisInsight .
  28. Web site: FIN 211 . AdisInsight .
  29. Web site: Phacetoperane - NLS Pharmaceutics Ltd . AdisInsight .
  30. Konofal E, Lecendreux M, Bizot JC, Lormier AT, Figadère B . NLS-3 (Levophacetoperane or (R,R) Phacetoperane): A Reverse Ester of Methylphenidate: A Literature Review . Curr Med Chem . 31 . 9 . 1069–1081 . 2024 . 36683369 . 10.2174/0929867330666230120161837 .
  31. Web site: Delving into the Latest Updates on LIT-001 with Synapse . Synapse . 2 August 2024 . 18 August 2024.
  32. Book: Nashar PE, Whitfield AA, Mikusek J, Reekie TA . Oxytocin . The Current Status of Drug Discovery for the Oxytocin Receptor . Methods Mol Biol . 2384 . 153–174 . 2022 . Springer . 34550574 . 10.1007/978-1-0716-1759-5_10 . 978-1-0716-1758-8 .
  33. Kaur H, Karabulut S, Gauld JW, Fagot SA, Holloway KN, Shaw HE, Fantegrossi WE . Balancing Therapeutic Efficacy and Safety of MDMA and Novel MDXX Analogues as Novel Treatments for Autism Spectrum Disorder . 2023 . Psychedelic Medicine . 1 . 3 . 166–185 . 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..
  34. PharmAla Biotech . Patent Application Published for PharmAla Biotech's MDMA Analogs . GlobeNewswire News Room . 23 February 2023 . 10 November 2024.
  35. PharmAla Biotech . PharmAla Biotech Signs Sale Agreement with Numinus . GlobeNewswire News Room . 26 March 2024 . 10 November 2024.
  36. Clark MR, Shaw HE, Fantegrossi WE . Poster 21: In vivo characterization of MBDB and its enantiomers in C57BL/6 and autism-like BTBR T+Itpr3tf/J mice . 16th Annual Behavior, Biology, and Chemistry: Translational Research in Substance Use Disorders, San Antonio, Texas, Embassy Landmark, 22-24 March 2024 . March 2024 .
  37. Web site: Psilocin - Pilz Bioscience . AdisInsight .
  38. Web site: QBM 001 . AdisInsight .
  39. Web site: Lesko . Lori . Biomarkers for autism . Drug Discovery News . 13 November 2018 . 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.”.
  40. Web site: Baeocystin - Pilz Bioscience . AdisInsight .
  41. Rakoczy RJ, Runge GN, Sen AK, Sandoval O, Wells HG, Nguyen Q, Roberts BR, Sciortino JH, Gibbons WJ, Friedberg LM, Jones JA, McMurray MS . Pharmacological and behavioural effects of tryptamines present in psilocybin-containing mushrooms . Br J Pharmacol . 181 . 19 . 3627–3641 . October 2024 . 38825326 . 10.1111/bph.16466 . free .
  42. Web site: Norpsilocin - Pilz Bioscience . AdisInsight .
  43. Web site: Roluperidone - Minerva Neurosciences/Mitsubishi Tanabe Pharma Corporation . AdisInsight .
  44. Web site: Squalamine - Enterin . AdisInsight .
  45. Web site: Psilocybin - COMPASS Pathways . AdisInsight .
  46. Web site: Research programme: autism therapeutic - Innovation Pharmaceuticals . AdisInsight .
  47. Web site: AB 1224 . AdisInsight .
  48. Web site: AGX 201 . AdisInsight .
  49. Web site: Aminolevulinic acid/sodium ferrous citrate - SBI Pharmaceuticals . AdisInsight .
  50. Web site: Aripiprazole transdermal - Aequus Pharmaceuticals . AdisInsight .
  51. Web site: BAER 101 . AdisInsight .
  52. Web site: BBP 472 . AdisInsight .
  53. Web site: Co-crystallised fasoracetam - Avalo Therapeutics . AdisInsight .
  54. Web site: Guanfacine once-daily - Takeda . AdisInsight .
  55. Web site: Research programme: autism spectrum disorder therapeutics - KoBioLabs . AdisInsight .
  56. Web site: NBTX 001 . AdisInsight .
  57. Web site: Oxytocin intranasal - OptiNose . AdisInsight .
  58. Web site: Research programme: allosteric modulators - Addex Therapeutics . AdisInsight .
  59. Web site: Research programme: antisense oligonucleotide therapeutics - RogCon U.R . AdisInsight .
  60. Web site: Research programme: autism and obesity therapeutics - Berand Neuropharmacology . AdisInsight .
  61. Web site: Research programme: brain development disorder therapeutics - Seaside Therapeutics . AdisInsight .
  62. Web site: Research programme: cannabinoid receptor modulators - GW Pharmaceuticals . AdisInsight .
  63. Web site: Research programme: cannabis extract therapeutics - Cannabis Science . AdisInsight .
  64. Web site: Research programme: central nervous system therapeutics - AbbVie/Rugen . AdisInsight .
  65. Web site: Research programme: CNS disorder therapeutics - Promentis Pharmaceuticals . AdisInsight .
  66. Web site: Research programme: CNS disorders therapeutics - Sage Therapeutics . AdisInsight .
  67. Web site: Research programme: G protein-coupled receptor modulating small molecules - Omeros Corporation . AdisInsight .
  68. Web site: Research programme: GPCR modulators - Nxera Pharma . AdisInsight .
  69. Web site: Research programme: immunomodulating bacteria-based therapeutics - 4D Pharma . AdisInsight .
  70. Web site: Research programme: metabotropic glutamate receptor 5 antagonists - Roche/Seaside Therapeutics . AdisInsight .
  71. Web site: Research programme: oxytocin intranasal - Pastorus Pharma . AdisInsight .
  72. Web site: Research programme: therapeutic autoantibodies - Sengenics . AdisInsight .
  73. Web site: RG 7713 . AdisInsight .
  74. Web site: Tideglusib - AMO Pharma . AdisInsight .
  75. Web site: Vafidemstat - Oryzon Genomics . AdisInsight .
  76. Web site: Balovaptan - Roche . AdisInsight .
  77. Web site: Blarcamesine - Anavex Life Sciences . AdisInsight .
  78. Web site: Brilaroxazine - Reviva Pharmaceuticals . AdisInsight .
  79. Web site: Carbetocin - Travere Therapeutics . AdisInsight .
  80. Web site: CX 516 . AdisInsight .
  81. Web site: EM 036 . AdisInsight .
  82. Web site: Fasoracetam - Avalo Therapeutics . AdisInsight .
  83. Web site: Fluoxetine-rapid dissolve - Autism Therapeutics . AdisInsight .
  84. Web site: GTS 21 . AdisInsight .
  85. Web site: Ketamine intranasal - Roivant Sciences . AdisInsight .
  86. Web site: Memantine - Children's Medical Center Corporation/Merz Pharma . AdisInsight .
  87. Web site: Oxytocin intranasal - Vyera Pharmaceuticals . AdisInsight .
  88. Web site: Research programme: AMPA receptor agonists - RespireRx . AdisInsight .
  89. Web site: Research programme: NMDA receptor modulators - AbbVie . AdisInsight .
  90. Web site: Risperidone extended release - Xbrane . AdisInsight .
  91. Web site: Secretin - 9 Meters Biopharma . AdisInsight .
  92. Web site: Suramin sodium - Bayer . AdisInsight .
  93. Web site: Trichuris suis ova - Fortress Biotech . AdisInsight .
  94. Web site: Trofinetide - ACADIA Pharmaceuticals/Neuren Pharmaceuticals . AdisInsight .
  95. Web site: Aripiprazole - Bristol-Myers Squibb/Otsuka Pharmaceutical . AdisInsight .
  96. Web site: Risperidone - Janssen . AdisInsight .
  97. Siafis S, Çıray O, Wu H, Schneider-Thoma J, Bighelli I, Krause M, Rodolico A, Ceraso A, Deste G, Huhn M, Fraguas D, San José Cáceres A, Mavridis D, Charman T, Murphy DG, Parellada M, Arango C, Leucht S . Pharmacological and dietary-supplement treatments for autism spectrum disorder: a systematic review and network meta-analysis . Mol Autism . 13 . 1 . 10 . March 2022 . 35246237 . 8896153 . 10.1186/s13229-022-00488-4 . free .
  98. Siegel M, Beaulieu AA . 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 . August 2012 . 22068820 . 10.1007/s10803-011-1399-2 .
  99. Aishworiya R, Valica T, Hagerman R, Restrepo B . An Update on Psychopharmacological Treatment of Autism Spectrum Disorder . Neurotherapeutics . 19 . 1 . 248–262 . January 2022 . 35029811 . 9130393 . 10.1007/s13311-022-01183-1 .
  100. Kisely S, Connor M, Somogyi AA, Siskind D . 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 . March 2023 . 35285280 . 10.1177/00048674221083868 .
  101. Danforth AL, Struble CM, Yazar-Klosinski B, Grob CS . MDMA-assisted therapy: A new treatment model for social anxiety in autistic adults . Prog Neuropsychopharmacol Biol Psychiatry . 64 . 237–249 . January 2016 . 25818246 . 10.1016/j.pnpbp.2015.03.011 . free .
  102. Chaliha D, Mamo JC, Albrecht M, Lam V, Takechi R, Vaccarezza M . A Systematic Review of the MDMA Model to Address Social Impairment in Autism . Curr Neuropharmacol . 19 . 7 . 1101–1154 . 2021 . 33388021 . 8686313 . 10.2174/1570159X19666210101130258 .
  103. Zhang S, Han F, Wang Q, Fan F . Probiotics and Prebiotics in the Treatment of Autism Spectrum Disorder: A Narrative Review . J Integr Neurosci . 23 . 1 . 20 . January 2024 . 38287844 . 10.31083/j.jin2301020 . free .
  104. He X, Liu W, Tang F, Chen X, Song G . Effects of Probiotics on Autism Spectrum Disorder in Children: A Systematic Review and Meta-Analysis of Clinical Trials . Nutrients . 15 . 6 . March 2023 . 1415 . 36986145 . 10054498 . 10.3390/nu15061415 . free .
  105. Markopoulos A, Inserra A, De Gregorio D, Gobbi G . Evaluating the Potential Use of Serotonergic Psychedelics in Autism Spectrum Disorder . Front Pharmacol . 12 . 749068 . 2021 . 35177979 . 8846292 . 10.3389/fphar.2021.749068 . free .
  106. Heifets BD, Salgado JS, Taylor MD, Hoerbelt P, Cardozo Pinto DF, Steinberg EE, Walsh JJ, Sze JY, Malenka RC . Distinct neural mechanisms for the prosocial and rewarding properties of MDMA . Sci Transl Med . 11 . 522 . December 2019 . 31826983 . 7123941 . 10.1126/scitranslmed.aaw6435 . FEN has been reported to improve social deficits in children with autism (34). However, like MDMA, long-term and/or heavy use of FEN is associated with cardiovascular and neurological toxicity (1, 7, 35)..
  107. Aman MG, Kern RA . Review of fenfluramine in the treatment of the developmental disabilities . J Am Acad Child Adolesc Psychiatry . 28 . 4 . 549–565 . July 1989 . 2670881 . 10.1097/00004583-198907000-00014 .
  108. Howes OD, Rogdaki M, Findon JL, Wichers RH, Charman T, King BH, Loth E, McAlonan GM, McCracken JT, Parr JR, Povey C, Santosh P, Wallace S, Simonoff E, Murphy DG . Autism spectrum disorder: Consensus guidelines on assessment, treatment and research from the British Association for Psychopharmacology . J Psychopharmacol . 32 . 1 . 3–29 . January 2018 . 29237331 . 5805024 . 10.1177/0269881117741766 .
  109. Gupta N, Gupta M, Gandhi R . Buspirone in Autism Spectrum Disorder: A Systematic Review . Cureus . 15 . 5 . e39304 . May 2023 . 37378184 . 10291962 . 10.7759/cureus.39304 . free .