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 (ASDs) and/or other pervasive developmental disorders (PDDs) 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 It is likely to become outdated with time.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
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 Cariprazine (Reagila, Symvenu, Vraylar; MP-214, RGH-188, WID-RGC20) – dopamine D2 and D3 receptor partial agonist, serotonin 5-HT1A receptor partial agonist, serotonin 5-HT2B receptor antagonist, and atypical antipsychotic Lumateperone (Caplyta) – dopamine D1 and D2 receptor antagonist, serotonin 5-HT2A receptor antagonist, α1-adrenergic receptor antagonist, serotonin reuptake inhibitor, and atypical antipsychotic Lurasidone (Latuda) – dopamine D2 and D3 receptor antagonist, serotonin 5-HT1A receptor partial agonist, serotonin 5-HT2A and 5-HT7 receptor antagonist, α2C-adrenergic receptor antagonist, and atypical antipsychotic Tasimelteon (Hetlioz) – melatonin MT1 and MT2 receptor agonist
Phase 2 AB-2004 – microbiome modulator Acamprosate (AOP-2020; Campral; SF-679/SF-775; SF-999) – unknown / GABAA receptor positive allosteric modulator and ionotropic glutamate NMDA receptor antagonist Alogabat (RG-7816, RG7816, RO-7017773; GABA-A-α5 PAM) – GABAA α5 subunit-containing receptor positive allosteric modulator Arbaclofen ((R)-baclofen; STX-209) – GABAB receptor agonist Cannabidiol (CBD; Epidiolex) – cannabinoid receptor modulator, other actions Cannabidiol transdermal patch/gel (Zygel; ZYN-002) – cannabinoid receptor modulator, other actions Cannabidivarin (CBDV; GWP-42006) – non-intoxicating cannabinoid receptor modulator, other actions CP-101 – bacteria relacement and gastrointestinal microbiome modulator JNJ-42165279 (JNJ-5279) – fatty acid amide hydrolase (FAAH) inhibitor Non-racemic MDMA (ALA-002; 70–80% (R)-MDMA, 20–30% (S)-MDMA) – serotonin, norepinephrine, and dopamine releasing agent, weak serotonin 5-HT2A, 5-HT2B, 5-HT2C receptor agonist, entactogen, and weak psychedelic hallucinogen Oxytocin (intranasal potentiated oxytocin; TI-001, TI-114, TNX-1900, TNX-2900) – oxytocin receptor agonist Pitolisant (Wakix; tiprolisant) – histamine H3 receptor inverse agonist Racemetirosine (DL-α-methyltyrosine; L1-79) – tyrosine hydroxylase inhibitor Suramin (IV suramin; PAX-101/PAX-102) – DNA synthesis inhibitor and anti-purinergic agent Zolmitriptan modified-release (ML-004, ML004) – serotonin 5-HT1B and 5-HT1D receptor agonist
Phase 1 (R)-MDMA ((R)-midomafetamine; MM-402) – serotonin, norepinephrine, and dopamine releasing agent and weak serotonin 5-HT2A, 5-HT2B, and 5-HT2C receptor agonist (entactogen and weak psychedelic hallucinogen) STP-1 (STALICLA Therapeutic Package 1) – phosphoric diester hydrolase inhibitor and sodium–potassium–chloride symporter inhibitor Sulforafan alfadex (sulforaphane alfadex; sulforafan α-cyclodextrin; SFX-01, STP-2; broccoli sprout extract) – various actions
Preclinical ASD-002 - a child-specific (non-acidic, sustained-release) anti-hyper excitatory ester prodrug of the potassium channel modulator NSAID mefenamic acid (MFA, approved as Ponstel) to prevent toddlers from becoming non-verbal (developmental language disorder, DLD)
Bryostatin-1 (MW-904) – protein kinase C stimulant CJRB-303 (CJRB-901, MRx-0006) – bacteria replacement Debamestrocel (growth factor-producing stem cell therapy) – dopaminergic cell replacement therapy FIN-211 – bacteria replacement and gastrointestinal microbiome modulator Levophacetoperane (NLS-3; (R,R)-phacetoperone; methylphenidate reverse ester) – norepinephrine and dopamine reuptake inhibitor and psychostimulant LIT-001 – small-molecule oxytocin receptor agonist MBDB – serotonin and norepinephrine releasing agent and weak serotonin 5-HT1 and 5-HT2 receptor ligand (entactogen) Psilocin (PLZ-1015) – non-selective serotonin receptor agonist and psychedelic hallucinogen QBM-001 – undefined mechanism of action (allosteric channel modulator; putative neuroprotective and anti-inflammatory agent)
Research Baeocystin (PLZ-1019) – non-hallucinogenic serotonin 5-HT2A receptor agonist, other actions Norpsilocin (PLZ-1017) – non-hallucinogenic serotonin 5-HT2A receptor agonist, other actions Roluperidone (CYR-101, MIN-101, MT-210) – serotonin 5-HT2A, sigma σ2, and α1A-adrenergic receptor antagonist Squalamine (ENT-01; Enterin-01; kenterin) – various actions
Phase unknown Psilocybin (COMP360; COMP-360) – non-selective serotonin receptor agonist and psychedelic hallucinogen
Not under development
Development suspended KM-391 – serotonin reuptake inhibitor
No development reported AB-1224 – microbiome modulator AGX-201 (histamine dihydrochloride salt) – histamine H1 receptor antagonist and histamine H3 receptor agonist Aminolevulinic acid/sodium ferrous citrate (5-ALA-SFC, 5-ALA/SFX; sodium ferrous citrate/aminolevulinic acid; SPP-003) – erythropoiesis stimulant and photosensitizer Aripiprazole transdermal (AQS-1301; transdermal aripiprazole) – dopamine D2 and D3 receptor partial agonist, serotonin 5-HT1A and 5-HT7 receptor partial agonist, serotonin 5-HT2A and 5-HT2B receptor antagonist or inverse agonist, and atypical antipsychotic BAER-101 (AZ-7325; AZD-7325) – selective GABAA α2 and α3 subunit-containing receptor positive allosteric modulator BBP-472 – phosphatidylinositol 3 kinase β (PI3Kβ) inhibitor Fasoracetam co-crystallised (co-crystallised fasoracetam; AEVI-004) – various actions and racetam Guanfacine once-daily (Guanfacine Carrier Wave; SPD-547) – α2-adrenergic receptor agonist KBLP-010 – bacteria replacement and microbiome modulator Oxytocin intranasal (OPN-300; OptiNose oxytocin) – oxytocin receptor agonist Research programme: allosteric modulators - Addex Therapeutics (various) – various actions Research programme: antisense oligonucleotide therapeutics - RogCon U.R (RCUR-313, RCUR-SMP) – voltage-gated sodium channel Nav1.2 expression stimulants Research programme: autism and obesity therapeutics - Berand Neuropharmacology – histone deacetylase inhibitors Research programme: brain development disorder therapeutics - Seaside Therapeutics (STX-110) – metabotropic glutamate mGlu5 receptor antagonists and muscarinic acetylcholine M1 receptor antagonists Research programme: cannabinoid receptor modulators - GW Pharmaceuticals (cannabigerol; CBG) – cannabinoid receptor modulators Research programme: cannabis extract therapeutics - Cannabis Science (CBIS compounds) – cannabinoid receptor modulators Research programme: central nervous system therapeutics - AbbVie/Rugen – undefined mechanism of action Research programme: CNS disorder therapeutics - Promentis Pharmaceuticals – antioxidants, glutamate receptor modulators, SLC7A11 modulators Research programme: CNS disorders therapeutics - Sage Therapeutics (SAGE-105; SGE-202; SGE-301; SGE-516) – GABAA receptor modulators and ionotropic glutamate NMDA receptor modulators Research programme: G protein-coupled receptor modulating small molecules - Omeros Corporation – G protein-coupled receptor modulator and neuromedin U receptor modulator Research programme: GPCR modulators - Nxera Pharma – various actions Research programme: immunomodulating bacteria-based therapeutics - 4D Pharma – bacteria replacements Research programme: metabotropic glutamate receptor 5 antagonists - Roche/Seaside Therapeutics – metabotropic glutamate mGlu5 receptor antagonists Research programme: oxytocin intranasal - Pastorus Pharma – neurotransmitter modulators/oxytocin receptor agonists Research programme: therapeutic autoantibodies - Sengenics – undefined mechanism of action RG-7713 (RG7713) – vasopressin V1A receptor antagonist Tideglusib (AMO-02, NP-031112, NP-12; Nypta, Zentylor) – glycogen synthase kinase 3β (GSK-3β) inhibitor Vafidemstat (ORY-2001) – dual lysine specific demethylase 1 (LSD1) inhibitor and monoamine oxidase B (MAO-B) inhibitor Xenon (NBTX-001) – ionotropic glutamate NMDA receptor antagonist
Discontinued Balovaptan (RG-7314, RO-5028442, RO-5285119) – vasopressin V1A receptor antagonist Blarcamesine (AE-37, ANA001, ANAVEX 2-73) – sigma σ1, muscarinic acetylcholine M1, and ionotropic glutamate NMDA receptor agonist Brilaroxazine (RP-5063, RP-5000) – dopamine D2, D3, D4 receptor partial agonist, serotonin 5-HT1A receptor agonist, serotonin 5-HT2A, 5-HT2B, 5-HT7 receptor antagonist, and atypical antipsychotic Bumetanide oral liquid (S-95008) – sodium–potassium–chloride symporter/cotransporter inhibitor and indirect GABAergic inhibitor discontinued after failed phase 3 trial Carbetocin (CYP-2001) – oxytocin receptor agonist CX-516 (1-BCP; BDP-12, SPD-420; AMPAlex) – AMPA receptor modulator EM-036 (memantine analogue) – ionotropic glutamate NMDA receptor antagonist, other actions Fasoracetam (AEVI-001, LAM-105, MDGN-001, NFC-1, NS-105) – various actions and racetam Fluoxetine rapid-dissolve (AT-001; AT001; NPL-2008; Serelsa; Zydis™ ODT fluoxetine) – serotonin reuptake inhibitor GTS-21 (DMXB-A, DMXB-A sustained release, DMXB-A-SR) – α7 nicotinic acetylcholine receptor partial agonist Ketamine intranasal (RVT-701) – ionotropic glutamate NMDA receptor antagonist Memantine (Namenda) – ionotropic glutamate NMDA receptor antagonist, other actions Oxytocin intranasal (Syntocinon Nasal Spray; TUR 001) – oxytocin receptor agonist Research programme: AMPA receptor agonists (ampakines, AMPAkines; CX compounds) - RespireRx – ionotropic glutamate AMPA receptor agonists Research programme: NMDA receptor modulators - AbbVie/Naurex (NRX-1050; NRX-1051; NRX-1059; NRX-105x; NRX-1060; NRX-2085; NRX-20xx) – ionotropic glutamate NMDA receptor modulators Risperidone extended-release (Risperisphere) – dopamine D2 and D3 receptor antagonist, serotonin 5-HT1B, 5-HT2A, 5-HT2C, and 5-HT7 receptor antagonist or inverse agonist, α1- and α2-adrenergic receptor antagonist, histamine H1 receptor inverse agonist, and atypical antipsychotic Secretin (INN-329, RG-1068; SecreFlo) – medical imaging enhancer (diagnosis) Suramin (Antrypol) – DNA-directed DNA polymerase inhibitor and intercellular signaling peptide/protein inhibitor Trichuris suis ova (CNDO-201, TSO, TSO-2500, TSO-7500) – immunomodulator Trofinetide (Daybue; G-2Me-PE; Glycyl-2-methyl-L-prolyl-L-glutamic acid; NNZ-2566; IGF-1 (1–3) analogue) – unknown / various actions
Formal development never or not yet started KNX-100 (KNX100; SOC-1) – oxytocin-like drug / indirect oxytocin receptor modulator
Clinically used drugs Approved drugs Aripiprazole (Abilify) – dopamine D2 and D3 partial agonist, serotonin 5-HT1A and 5-HT7 receptor partial agonist, serotonin 5-HT2A and 5-HT2B receptor antagonist or inverse agonist, and atypical antipsychotic Risperidone (Risperdal) – dopamine D2 and D3 receptor antagonist, serotonin 5-HT1B, 5-HT2A, 5-HT2C, and 5-HT7 receptor antagonist or inverse agonist, α1- and α2-adrenergic receptor antagonist, histamine H1 receptor inverse agonist, and atypical antipsychotic
Off-label drugs α2-Adrenergic receptor agonists (e.g., clonidine, guanfacine) Anticonvulsants/mood stabilizers (e.g., valproic acid, lamotrigine) Antipsychotics (non-licensed) (e.g., haloperidol, olanzapine) Cannabinoids (e.g., cannabis, dronabinol, nabilone) Dietary supplements (e.g., N-acetylcysteine, omega-3 fatty acids, sulforaphane)