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ambroxol (ambroxol, Diffucaps)

✓ Approved

Adare Pharma Solutions · Small Molecule · Small Molecule

What is ambroxol?

ambroxol is a small molecule developed by Adare Pharma Solutions. It is approved for therapeutic indications via oral (po).

Drug Profile

Brand Namesambroxol, Diffucaps
CompanyAdare Pharma Solutions
Drug ClassSmall Molecule
RouteOral (PO)
StatusApproved
Sources: ClinicalTrials.gov, Adare Pharma Solutions

Therapeutic Indications

ambroxol is developed for 1 unique indication across 1 therapeutic area.

Therapeutic AreaConditionPhase
Respiratory, thoracic and mediastinal disordersBronchitis chronic✓ Approved

Related Research Articles

PubMedMolecular biology reports2026-08-21

Glucocerebrosidase dysfunction in GBA1 carriers: insights from blood and macrophage analyses.

Nikolaev Mikhail M, Kopytova Alena A, Izyumchenko Artem A, Senkevich Konstantin K et al.

Mutations in the GBA1 gene, which encodes the lysosomal enzyme glucocerebrosidase (GCase), are the most common genetic factor associated with Parkinson's disease (PD). These mutations are classified as "severe" or "mild" based on the residual GCase activity. This study aimed to compare the biochemical characteristics of peripheral blood and macrophages derived from peripheral blood mononuclear cells (PBMC-derived macrophages) from PD patients with GBA1 mutations (GBA1-PD) and healthy GBA1 mutation carriers (GBA1-carriers). 31 GBA1-PD patients, 24 GBA1-carriers and 247 controls were enrolled. We assessed GCase activity, levels of the lysosphingolipid hexosylsphingosine (HexSph) and the proteins GCase, alpha-synuclein, and cathepsin D as well as GCase translocation to lysosomes in PBMC-derived macrophages. Biochemical analysis of PBMC-derived macrophages revealed similar impairments in GCase activity, lysosomal translocation, elevated HexSph and alpha-synuclein, and decreased cathepsin D in both GBA1-PD and GBA1-carriers compared to controls. However, when the mutations were divided according to their severity, almost all differences were only observed in the carriers of "severe" mutations. The ratio of GCase activity to HexSph level in blood significantly differed between GBA1-PD and GBA1-carriers. Additionally, we retrospectively analyzed data on GCase activity and HexSph level in blood of GBA1-PD patient during the presymptomatic period prior motor symptom onset, following oral ambroxol treatment (1200 mg per day). Biochemical alterations in GCase function are linked to GBA1 mutations independent of PD status, with "severe" mutations notably impacting lysosomal function and multiple cellular processes. We propose the GCase activity to HexSph ratio as a novel biomarker for disease progression and monitoring therapeutic response.

PMID 42627573
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PubMedFrontiers in pediatrics2026-08-18

Analysis of the interventional effects of ambroxol hydrochloride and ontelukast sodium combined with azithromycin on immune function and C-reactive protein serum expression in children with severe Mycoplasma pneumoniae pneumonia.

Liu Xueru X, Chen Xiang X, Ye Shan S

The aim of the study was to investigate the clinical effects of ambroxol hydrochloride and montelukast sodium combined with azithromycin on immune function, inflammatory cytokines, and pulmonary function in children with severe Mycoplasma pneumoniae pneumonia (SMPP). A retrospective cohort study was conducted on 103 children with SMPP treated at Wuhan Children's Hospital, Tongji Medical College, Huazhong University of Science & Technology, from February 2023 to February 2025. On the basis of different treatment regimens recorded in the electronic medical record system, the children were divided into a control group (n = 50, montelukast sodium combined with azithromycin) and an observation group (n = 53, ambroxol hydrochloride added to the control regimen). To address the selection bias inherent in the retrospective design, a propensity score analysis based on inverse probability of treatment weighting (IPTW) was performed; a logistic regression model incorporating age, sex, disease duration, only-child status, paternal and maternal education levels, and place of residence as covariates was used to derive the propensity scores, and stabilized weights were applied so that all 103 patients contributed to the weighted analysis without exclusion. Clinical data were collected and compared, including baseline characteristics, symptoms and signs (time for cough to subside, time for body temperature to normalize, time for sputum to disappear, and time for rales to disappear), immune function (CD3+, CD4+, CD8+, CD4+/CD8+), inflammatory cytokines [tumor necrosis factor-alpha (TNF-α), interferon-gamma (IFN-γ), interleukin-2 (IL-2), interleukin-4 (IL-4), and C-reactive protein (CRP)], pulmonary function [forced expiratory volume in the first second (FEV1), forced vital capacity (FVC), and maximal mid-expiratory flow (MMEF)], clinical efficacy, and adverse reactions. The total clinical efficacy rate was pre-specified as the primary outcome, and all remaining endpoints were treated as secondary or exploratory; a Bonferroni-corrected significance threshold of α = 0.003 was applied across the 16 secondary continuous outcomes. Effect sizes (Cohen's d) and 95% confidence intervals (CIs) were computed for all between-group comparisons. After treatment, the observation group showed significantly greater improvement in symptoms and signs, immune function, inflammatory cytokines, and pulmonary function compared with the control group (all P < 0.05). The total clinical efficacy rate of the observation group was significantly higher than that of the control group [96.23% vs. 82.00%; χ 2 = 5.459, P = 0.019; odds ratio (OR) = 5.59, 95% CI: 1.14-27.38; the wide confidence interval, driven by the small number of ineffective cases, indicates that the magnitude of the efficacy advantage should be interpreted with caution]. The total incidence of adverse reactions was comparable between the observation group and the control group (11.32% vs. 10.00%; χ 2 = 0.047, P = 0.828), indicating acceptable safety. The triple combination of ambroxol hydrochloride, montelukast sodium, and azithromycin is associated with improved inflammatory status, enhanced immune function, and better pulmonary function in children with SMPP, with a favorable safety profile. These findings warrant confirmation in prospective, multicenter randomized controlled trials.

PMID 42609515
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PubMedInfection and drug resistance2026-07-22

Association of Ambroxol Hydrochloride and Clenbuterol Hydrochloride Oral Solution with Respiratory Symptom Improvement and Safety in Chinese Children with Pneumonia: A Real-World Propensity Score-Matched Study.

Liang Ying Y, Ling Yesheng Y, Hu Bing B, Zou Yingxue Y et al.

To evaluate the association of ambroxol hydrochloride and clenbuterol hydrochloride oral solution (AHCHOS) with respiratory symptom improvement and safety in Chinese children with pneumonia using real-world data. A propensity score-matched (PSM) cohort study was conducted. Children patients (≤14 years) with a diagnosis of pneumonia between May, 2018 and July, 2019 were considered as the study population. The main outcome of interest was the overall rate of improvement of respiratory symptoms in treatment groups with or without AHCHOS at day 7 using respiratory symptom scores (QS) and Visual Analog Scale (VAS) score of severity of respiratory signs. Secondary end points include medication adherence assessment and safety assessment. A total of 3103 children with a diagnosis of pneumonia were included. After propensity score matching, a sample of 1428 patients was analyzed. AHCHOS use was associated with greater improvement in cough score (p=0.01) and day 7 clinical sign improvement rate (p=0.03) compared with no AHCHOS use. Exploratory subgroup analyses suggested larger differences in children aged 3-6 years and in selected severity strata. Medication adherence assessmentdid not differ significantly between groups at days 4, 7, 14, and 28. Adverse events were similar between groups. In this real-world propensity score-matched analysis, AHCHOS use was associated with improved respiratory symptom outcomes in children with pneumonia and appeared to have an acceptable safety profile. These findings should be interpreted as observational and hypothesis-generating. The study was registered at Chinese Clinical Trial Registry (https://www.chictr.org.cn/, ChiCTR1800015818).

PMID 42483308
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PubMedTranslational pediatrics2026-07-11

Efficacy and safety of inhaled ambroxol hydrochloride solution in Chinese pediatric patients with acute lower respiratory tract infections: a real-world, multicenter, open-label, single-arm study.

Wang Hao H, Zou Yingxue Y, Shang Yunxiao Y, Chen Mingwu M et al.

Although randomized controlled trials (RCTs) have confirmed the mucolytic efficacy of ambroxol hydrochloride solution for inhalation (AHSI) in selected cohorts, their stringent exclusion criteria often omit children with comorbidities and complex presentations encountered in routine practice. Consequently, real-world evidence is needed to evaluate the effectiveness and safety of AHSI in a broader, clinically representative pediatric population with acute lower respiratory tract infections (ALRTIs). This study therefore aimed to assess the real-world effectiveness, safety, and nebulizer compatibility of a 7-day AHSI regimen added to standard care in a large, multicenter cohort of hospitalized pediatric patients with ALRTI. This real-world, multicenter, open-label, single-arm study enrolled hospitalized patients aged ≥6 months with ALRTI (acute bronchitis, bronchiolitis, or pneumonia) and symptom duration <7 days across 62 centers in China (April 2021-April 2022). Key inclusion criteria included a cough score ≥2 (0-4 scale), tenacious sputum, and difficulty expectorating. Major exclusions comprised severe pneumonia, bronchial asthma, interstitial lung disease, significant hepatic or renal dysfunction [alanine aminotransferase (ALT) >1.5× upper limit of normal (ULN), total bilirubin (TBil) or serum creatinine (Scr) > ULN], other severe comorbidities, known hypersensitivity to ambroxol, or recent trial participation. Participants received weight-based doses of nebulized AHSI twice daily for 7 days as add-on to standard care. Follow-up visits occurred at day 4 and day 7 (end of treatment). Primary endpoints were the cough improvement rate (defined by a reduction in cough score) and the overall clinical response rate (investigator-assessed improvement). Secondary endpoints included changes from baseline in cough, throat rales, and pulmonary auscultation scores. Safety assessments comprised monitoring of adverse events (AEs) (coded with MedDRA), vital signs, and laboratory tests (hematology, biochemistry, urinalysis) at baseline and day 7. A total of 2,599 children were enrolled [full analysis set (FAS)]. At baseline, mean age was 3.60±2.50 years, 57.6% were male, and symptom scores were: cough 2.10±0.30, throat sputum 1.65±0.59, lung auscultation 1.44±0.70. In the FAS, the cough improvement rate was 96.73% [95% confidence interval (CI): 96.05-97.41] and the clinical response rate was 94.73% (95% CI: 93.87-95.59). All symptom scores decreased significantly from baseline to day 7 (P<0.001). Drug-related AEs (DRAEs) occurred in 0.39% of patients, predominantly mild-to-moderate rash, transient liver enzyme elevations, and gastrointestinal events; no serious DRAEs were reported. Outcomes were consistent across pneumonia and bronchitis subgroups and across various nebulizer types. This large real-world study demonstrated that a 7-day course of AHSI, added to standard care, was associated with clinically meaningful improvements in respiratory symptoms and a favorable safety profile in children with ALRTI. The consistent effects across disease subtypes and nebulizer devices underscore the practical utility of AHSI in diverse pediatric settings. While the single-arm design limits causal inference, these findings provide robust real-world evidence supporting AHSI as an effective expectorant option. Prospective confirmation through RCTs will further define its role in first-line therapy.

PMID 42433940
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PubMedWater research2026-07-09

Photodegradation of expectorant drug ambroxol in seawater environment: Seasonal impact on a global scale.

Zhang Ying Y, Li Xiaoci X, Chovelon Jean-Marc JM, Ji Yuefei Y et al.

The ocean is a globally important zone for the photochemical transformation of various organic contaminants. Its unique chemical composition, especially the high concentration of halide ions, can significantly alter the photochemical reactivity and fate of these contaminants. Herein, we systematically investigated the effects of marine constituents on the sunlight-driven photolysis of ambroxol (AMB), an expectorant drug extensively used following COVID-19 pandemic, and assessed its spatiotemporal persistence on a global scale. The results revealed that marine matrix significantly promoted AMB photodegradation in a halide-specific manner, where chloride drove such acceleration via photonucleophilic substitution, while bromide exerted an inhibitory effect. Differing from the pathways in pure water, the marine photolysis of AMB was dominated by photonucleophilic substitution, generating the primary chlorinated product with a substantial formation yield of ∼50.5%. Toxicity evaluation suggested that the halogen exchange reaction reduced the toxicity of the photoproducts, whereas other transformation pathways heightened the environmental risks of AMB. Utilizing the global-scale photolysis model GCSOLAR, this study further indicated that near-surface environmental persistence of AMB was strictly governed by geographic latitude and seasonal dynamics, where environmental half-lives remained minimal (1.24-1.83 h) in equatorial zones while rising to 34.77 h in high-latitude regions during winter. These findings highlighted the critical role of the marine matrix and global spatiotemporal variations in collectively reshaping the transformation landscape and intensifying the ecological risks of typical emerging contaminants.

PMID 42419117
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PubMedEuropean respiratory review : an official journal of the European Respiratory Society2026-06-25

Mucoactive agents in bronchiectasis: a systematic review and meta-analysis.

McCullough Benjamin B, Busby John J, O'Neill Brenda B, Connolly Bronwen B et al.

Mucoactive agents aim to improve mucociliary clearance in bronchiectasis, disrupting the cycle of impaired clearance, infection and inflammation. Registry data show 28% of patients use these agents, but prescribing varies due to limited evidence. We aimed to update evidence on mucoactive agents' effects in adults with bronchiectasis. We conducted a systematic review and meta-analysis, searching Medline, Embase, CENTRAL and trial registries to 1 October 2025. Eligible studies included adults with bronchiectasis, evaluating any mucoactive agent versus placebo or control. Cystic fibrosis and paediatric studies were excluded. The primary outcome was exacerbation frequency; secondary outcomes included lung function, quality of life and adverse events. From 1512 records, 24 studies (20 randomised, four observational: n=7051) evaluated eight mucoactive agents (ambroxol, bromhexine, carbocisteine, erdosteine, hypertonic saline, mannitol, N-acetylcysteine, rhDNase). Most trials had high or some risk of bias. Seven randomised studies (n=1259) reported exacerbations; pooled analysis did not demonstrate a statistically significant difference in annualised exacerbation incidence (mean difference -0.40 per patient per year, 95% CI -1.04-0.24; p=0.22; I2=91%; very low certainty). Nine studies (n=767) reported percent predicted forced expiratory volume in 1 s (FEV1 % pred), showing a small mean increase of 3.23% (95% CI 0.31-6.15; p=0.03; I2=69.9%; very low certainty). Pooled analyses did not demonstrate significant differences for other spirometry measures, quality of life or adverse events. Pooled evidence did not show a reduction in exacerbations and FEV1 % pred improvements were small. Evidence certainty was low/very low, meaning overall clinical benefit remains uncertain, highlighting the need for targeted trials of specific agents and subgroups.

PMID 42342264
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