Drug Database
AC

acetylcysteine

✓ Approved

Cumberland Pharmaceuticals Inc · Small Molecule · Small Molecule

What is acetylcysteine?

acetylcysteine is a small molecule developed by Cumberland Pharmaceuticals Inc. It is approved for therapeutic indications via injectable (others) or intravenous (iv).

Drug Profile

CompanyCumberland Pharmaceuticals Inc
Drug ClassSmall Molecule
RouteInjectable (Others), Intravenous (IV)
StatusApproved
Sources: ClinicalTrials.gov, Cumberland Pharmaceuticals Inc

Therapeutic Indications

acetylcysteine is developed for 2 unique indications across 2 therapeutic areas.

Therapeutic AreaConditionPhase
Hepatobiliary disordersHepatic function abnormal✓ Approved
Injury, poisoning and procedural complicationsToxicity to various agents✓ Approved

Related Research Articles

PubMedInfection and drug resistance2026-09-19

The Auranofin-Colistin Combination Efficiency Against the Gram-Negative Strains: A Review.

Ionescu Mihaela Ileana MI

Multidrug-resistant bacteria that possess resistance genes against last-resort antibiotics, such as colistin, are notably prevalent in hospital environments. Therapeutic options for addressing these infections are limited, prompting researchers to explore alternative strategies. One promising approach is drug repurposing, which involves testing combinations of antibiotics with other medications to discover potential synergistic effects against multidrug-resistant (MDR) strains. Auranofin, an antirheumatic drug known for its anti-inflammatory and antineoplastic properties, has recently been investigated in combination with colistin as a potential therapeutic strategy for treating infections caused by multidrug-resistant bacteria. A review of 10 articles retrieved from five public scientific databases indicates that all authors reported synergistic activity of the auranofin-colistin combination against multidrug-resistant (MDR) species, with effectiveness demonstrated in both in vitro and in vivo experiments. However, the minimal inhibitory concentrations (MICs) of this combination vary, and the precise mechanism of action remains unclear. Recent studies examining the crystal structure of metallo-beta-lactamase type 2 (NDM-1) and a probable phosphatidylethanolamine transferase, MCR-1 (which confers mobilized colistin resistance), suggest that activation with glutathione or N-acetylcysteine is necessary for efficacy. Additionally, there is strong evidence indicating that auranofin acts as a dual inhibitor of both MCR and metallo-beta-lactamase (MBL) by displacing the Zn(II) cofactor.

PMID 42761945
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PubMedExperimental lung research2026-09-18

Mechanism of IH promoting pulmonary arterial hypertension through ROS-NLRP3 inflammasome mediated endothelial cell pyroptosis.

Xu Yangxia Y, Pang Xiue X

To investigate the mechanism by which Intermittent Hypoxia (IH) promotes Pulmonary Arterial Hypertension (PAH), focusing on ROS-NLRP3 inflammasome-mediated endothelial cell pyroptosis. Human Pulmonary Microvascular Endothelial Cells (HPMECs) and a monocrotaline-induced PAH rat model were exposed to an IH environment. Interventions included the ROS inhibitor N-acetylcysteine (NAC) and the NLRP3 inhibitor MCC950. Assessments measured cell survival, LDH activity, ROS levels, apoptosis, inflammatory cytokines (IL-1β, IL-18), and protein expression of NLRP3 inflammasome components. In rats, hemodynamics, right ventricular hypertrophy, and pulmonary vascular remodeling were evaluated. IH exposure significantly decreased HPMEC survival and increased LDH activity, ROS levels, apoptosis rate, and the expression of NLRP3, Caspase-1, and ASC proteins. These effects, along with elevated IL-1β and IL-18, were reversed by NAC and MCC950 treatment. In PAH rats, IH exacerbated right ventricular systolic pressure, pulmonary artery pressure, vascular remodeling, and serum inflammatory markers, which were similarly ameliorated by NAC and MCC950. IH promotes the progression of PAH by inducing endothelial cell pyroptosis through the activation of the ROS-NLRP3 inflammasome pathway.

PMID 42757872
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PubMedChemico-biological interactions2026-09-18

Fluoride-Induced Testicular Spermatogenic Dysfunction: The Pivotal Role of the AIM2-Mediated Caspase-3/GSDME Pyroptosis Pathway.

Wang Guoqing G, Ba Ruijie R, Liu Bin B, Wang Yan Y et al.

Endemic fluorosis from high-fluoride groundwater is a global concern. While fluoride exposure is linked to male infertility, the mechanisms remain unclear. This study utilized male Sprague-Dawley (SD) rats and in vitro immortalized human testicular Sertoli (iHTS) cells to investigate the potential mechanisms underlying sodium fluoride (NaF)-induced testicular damage. In vivo, three-month NaF exposure caused testicular tissue disorganization, decreased sperm viability and motility, and increased sperm abnormalities. Furthermore, NaF exposure significantly altered the oxidative stress status in the rat testis, evidenced by decreased total antioxidant capacity (T-AOC), total superoxide dismutase (T-SOD), and glutathione (GSH) levels, along with increased malondialdehyde (MDA) content. Meanwhile, levels of pyroptosis-related proteins (AIM2, Cleaved Caspase-3, GSDME, and GSDME-N), as well as LDH, interleukin (IL)-1β, and IL-18, were significantly increased. Similar results were observed in vitro. Interestingly, knocking down AIM2 in NaF-treated iHTS cells significantly decreased the levels of Cleaved Caspase-3, GSDME, GSDME-N, IL-1β, IL-18, and LDH. Furthermore, inhibiting NaF-induced oxidative stress with N-acetylcysteine (NAC) effectively mitigated these NaF-induced protein and biochemical alterations. These findings suggest that excessive fluoride exposure induces oxidative stress in testicular tissue and cells, subsequently activating the AIM2/Caspase-3/GSDME pyroptotic axis, thereby leading to testicular damage.

PMID 42759637
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PubMedCellular signalling2026-09-18

Low-dose polysaccharides exert dual redox effects via the ROS-mediated MAPK pathway: Antioxidant alone or pro-oxidant when GLP is combined with doxorubicin in A2780 ovarian cancer cells.

Zhang Yuqian Y, Li Xinru X, Shen Yutong Y, Yang Lijuan L et al.

Ovarian cancer (OC) is a lethal gynecological malignancy. Patients usually consume low doses of natural polysaccharides through their diet as exogenous antioxidants. This study aims to explore the dual effects of low doses of five natural polysaccharides (GLP, LBP, LNT, APS, FUC) on A2780 OC cells, as well as the underlying molecular mechanisms. The redox effects of their individual actions and the combined action of GLP and doxorubicin (DOX) were evaluated by ROS, malondialdehyde (MDA), superoxide dismutase (SOD) and glutathione (GSH) assays; their growth/apoptosis effects were assessed by MTT, colony formation and flow cytometry. Network pharmacology predicted the MAPK pathway, which was verified by Western blot. In vivo, a xenograft model was established to evaluate the effect of the combination of GLP and DOX. The results showed that low doses of polysaccharides reduced ROS levels, promoted cell viability and colony-forming capacity, upregulated p-ERK levels, and downregulated p-p38 and p-JNK. In vitro, the same dose of GLP combined with DOX increased ROS levels, inhibited cell growth, induced apoptosis, and activated all three MAPK subfamilies. The ROS scavenger N-acetylcysteine (NAC) reversed these effects. In vivo, GLP combined with DOX synergistically inhibited tumor growth. Low doses of natural polysaccharides have specific dual effects: when used alone, they have antioxidant effects, but when GLP is combined with DOX, they have pro-oxidant and tumor-suppressive effects. Both of these effects are mediated through the ROS-MAPK pathway. This provides an experimental basis for mechanism studies and clinical applications of chemotherapy combined with polysaccharides in epithelial OC.

PMID 42759906
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PubMedAmerican journal of medical genetics. Part A2026-09-17

L-Cysteine and N-Acetylcysteine Supplementation Improves Clinical Outcome in a Patient With COXPD10.

Pillai Nishitha R NR, Elsbecker Sara A SA, McCarthy Grace Bronken GB, Weisshappel Kaitlin K et al.

MTO1 is a nuclear gene that encodes a mitochondrial protein essential for modifying mitochondrial transfer RNAs (tRNAs) and stabilizing codon-anticodon interactions to ensure accurate and efficient mitochondrial protein synthesis and oxidative phosphorylation. Mitochondrial tRNA translation optimization 1 (MTO1) plays an important role in the mitochondrial tRNA taurinomethylation modification by using the amino acid taurine, obtained from cysteine metabolism, at the wobble position U34 of the anticodon loop. Biallelic pathogenic variants in MTO1 cause combined oxidative phosphorylation deficiency 10 (COXPD10) (OMIM#614702). In the severe end of the spectrum, COXPD10 is characterized by infantile-onset hypertrophic cardiomyopathy and lactic acidosis with perinatal mortality when associated with nonsense and frameshift variants. The extra cardiac phenotypes include muscle hypotonia, feeding difficulties, psychomotor delay, optic atrophy, encephalopathy, and seizures. Currently, there is no targeted treatment for this condition aside from supportive care. Herein, we report a 22-month-old child, diagnosed early with a genotype predictive of severe COXPD10, who was initiated on treatment with L-cysteine and N-acetylcysteine (NAC) early in life and did not develop cardiac manifestations. This outcome suggests a potential benefit and improved clinical outcome with early disease-specific treatment initiation.

PMID 42753170
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PubMedFrontiers in pediatrics2026-09-17

Fetal and neonatal effects of N-acetylcysteine for maternal chorioamnionitis: a systematic review.

Elgharbawy Fawzia Mohamed FM, Bayoumi Mohammad A A MAA, Ajele Kenni W KW, Lwaleed Bashir A BA

Maternal chorioamnionitis (mCA) and intra-amniotic infection/inflammation (Triple I) are major causes of fetal inflammatory injury and are associated with preterm birth, white matter injury, cerebral palsy, bronchopulmonary dysplasia (BPD), and necrotising enterocolitis (NEC). No established therapy directly targets the inflammatory and oxidative pathways underlying fetal neuroinjury. N-acetylcysteine (NAC), a glutathione precursor with antioxidant and anti-inflammatory properties, has emerged as a potential adjunctive neuroprotective therapy, although human evidence remains limited. To evaluate fetal and neonatal outcomes associated with antenatal or intrapartum NAC administration in pregnancies complicated by mCA or Triple I. A systematic review was conducted using MEDLINE, Embase, Cochrane CENTRAL, and ClinicalTrials.gov from inception to December 23, 2025. Eligible studies included randomised controlled trials, prospective cohorts, non-randomised studies, and translational animal models evaluating NAC exposure in mCA or Triple I. Clinical outcomes, biomarkers, pharmacokinetics, and mechanistic pathways were synthesised narratively. Risk of bias was assessed using RoB 2, ROBINS-I, and SYRCLE tools. Ten studies met the inclusion criteria, including five human and six translational animal studies. Human evidence involved approximately 143 mother-infant dyads. NAC exposure was associated with preserved cerebrovascular coupling, improved delivery-room adaptation, reduced severe neonatal morbidity, and lower BPD rates without major safety concerns. Pharmacokinetic studies demonstrated rapid placental transfer and gestation-dependent neonatal clearance. Animal studies consistently showed reductions in inflammatory cytokines, oxidative stress, apoptosis, and microstructural brain injury. NAC demonstrates biologically plausible neuroprotective potential in mCA and Triple I; however, the evidence remains preliminary due to small sample sizes and heterogeneous protocols. However, some studies showed no or negative effects. Large multicenter randomised trials with standardised dosing and long-term neurodevelopmental follow-up are needed before routine clinical implementation can be recommended.

PMID 42751421
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