This is a list of investigational myalgic encephalomyelitis/chronic fatigue syndrome drugs, or drugs that are currently under development for clinical use for the treatment of myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS) but are not yet approved. Chemical/generic names are listed first, with developmental code names, synonyms, and brand names in parentheses. The format of list items is "Name (Synonyms) – Mechanism of Action [Reference]". This list was last comprehensively updated in September 2025. It is likely to become outdated with time.
Under development
Phase 2/3 Duloxetine (Ariclaim; Cymbalta; LY-227942; LY-248686; Xeristar; Yentreve) – serotonin–norepinephrine reuptake inhibitor (SNRI) [1]
Phase 2 Bocidelpar (ASP-0367; MA-0211; MTB-1) – peroxisome proliferator-activated receptor δ (PPARδ) modulator [2] OSU-6162 (PNU-9639; PNU-96391; PNU-96391A) – serotonin 5-HT2A receptor partial agonist (non-hallucinogenic), dopamine D2 receptor partial agonist, and sigma σ1 receptor ligand (so-called "monoaminergic stabilizer")
Phase 1/2 CT-38 (CT38) – CRF receptor type 2 modulator [3]
Preclinical Flmodafinil (CRL-40,940; NLS-14; NLS-4; JBG01-41; bisfluoromodafinil; lauflumide) – selective atypical dopamine reuptake inhibitor [4]
Not under development
No development reported Celecoxib/famciclovir (IMC-1) – combination of celecoxib (COX-2 inhibitor/NSAID) and famciclovir (antiviral) [5] Methylphenidate (KPAX-002) – norepinephrine–dopamine reuptake inhibitor (NDRI) [6] Midodrine (Amatine; Gudon; Gutron; Metligine; Midon; ProAmatine; ST-1085; TS-701) – α1-adrenergic receptor agonist [7] Rovunaptabin (BC-007) – oligonucleotide aptamer (binds to and neutralizes autoantibodies against GPCRs) [8] Valganciclovir (Cymeval; R127; RG-127; RO-1079070/194; RS-079070194; RS-79070; TA-9070; Valcyte; Valgancyclovir; Valixa) – antiviral [9]
Discontinued Alpha-1 antitrypsin modified process (Alpha-1 MP; Lynspad; Prolastin; Prolastina; Pulmolast; TAL-6004) – immunomodulator and serine endopeptidase inhibitor [10] Droxidopa (L-DOPS; Threo-DOPS; Northera) – norepinephrine prodrug and non-selective adrenergic receptor agonist [11] Galantamine (GP-37267; Nivalin; R-113675; Razadyne; Reminyl) – acetylcholinesterase inhibitor [12] Research programme: androgen-based therapeutics - CPEX – androgen receptor agonists [13] SPV-30 – undefined mechanism of action [14]
Formal development never or not yet started Solriamfetol (Sunosi) – norepinephrine–dopamine reuptake inhibitor
Clinically used drugs
Approved drugs Rintatolimod (AMP-516; AMP-518; Ampligen; Atvogen; Rintamod; PolyI:PolyC12U) – mismatched double-stranded polymer of RNA (dsRNA) and toll-like receptor 3 (TLR3) agonist – only approved and available in Argentina [15]
Off-label drugs Analgesics (e.g., acetaminophen, NSAIDs) Antidepressants (e.g., SSRIs, SNRIs, TCAs, MAOIs, mirtazapine, bupropion, trazodone) Antivirals (e.g., inosine pranobex) Atypical antipsychotics (e.g., aripiprazole) Corticosteroids (e.g., hydrocortisone, fludrocortisone) Gabapentinoids (α2δ voltage-dependent calcium channel ligands) (e.g., gabapentin, pregabalin) Immunotherapy (e.g., rituximab, interferon, immunoglobulin) Naltrexone (low-dose) Sleep aids (e.g., antihistamines, orexin receptor antagonists, Z-drugs, benzodiazepines, trazodone, melatonin) Stimulants (e.g., amphetamine, lisdexamfetamine, methylphenidate) Sympathomimetics (e.g., phenylephrine) Vitamins, minerals, and dietary supplements (e.g., NADH, coenzyme Q10, carnitine, omega fatty acids, magnesium, vitamin B12) Wakefulness-promoting agents (e.g., modafinil)
See also List of investigational drugs List of investigational fatigue drugs List of investigational long COVID drugs List of investigational fibromyalgia drugs List of investigational orthostatic intolerance drugs Management of ME/CFS
References
External links AdisInsight - Springer
