• Source: List of investigational autism and pervasive developmental disorder drugs

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 It is likely to become outdated with time.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




= 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
Bumetanide oral liquid (S-95008) – sodium–potassium–chloride symporter/cotransporter inhibitor and indirect GABAergic inhibitor
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
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

=
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
NBTX-001 – ionotropic glutamate NMDA receptor antagonist
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 and dissociative hallucinogen NBTX 001 - AdisInsight




= 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
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




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)
Dietary supplements (e.g., N-acetylcysteine, omega-3 fatty acids, sulforaphane)
Entactogens (serotonin releasing agents) (e.g., MDMA)
Melatonin receptor agonists (e.g., melatonin)
Norepinephrine reuptake inhibitors (NRIs) (e.g., atomoxetine)
Opioid receptor antagonists (e.g., naltrexone)
Other antidepressants (e.g., mirtazapine)
Oxytocin receptor agonists (e.g., oxytocin)
Probiotics and prebiotics
Psychostimulants (norepinephrine–dopamine releasing agents and/or reuptake inhibitors) (e.g., amphetamine, methylphenidate)
Selective serotonin reuptake inhibitors (SSRIs) (e.g., fluoxetine, fluvoxamine, sertraline, citalopram)
Serotonergic psychedelics (e.g., psilocybin, lysergic acid diethylamide (LSD))
Serotonin–norepinephrine reuptake inhibitors (SNRIs) (e.g., milnacipran)
Serotonin releasing agents (e.g., fenfluramine—withdrawn and no longer recommended)
Serotonin 5-HT1A receptor agonists (e.g., buspirone)
Tricyclic antidepressants (TCAs) (e.g., clomipramine)




See also


List of investigational drugs
List of investigational social anxiety disorder drugs




References






External links


AdisInsight - Springer

Kata Kunci Pencarian:

• List of investigational autism and pervasive developmental disorder drugs
• Lists of investigational drugs
• List of investigational social anxiety disorder drugs
• List of investigational hallucinogens and entactogens
• Attention deficit hyperactivity disorder
• (R)-MDMA
• Social anxiety disorder
• Donepezil
• Obsessive–compulsive disorder
• Antipsychotic