Drug Database
AM

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

PubMedFrontiers in pharmacology2026-10-09

Incidence, burden, and associated factors of severe potential drug-drug interactions in intensive care unit patients: a real-world retrospective study.

Jiang Huaqiao H, Shen Tingting T, Yang Yun Y, Zhang Lin L et al.

Severe potential drug-drug interactions (pDDIs) in intensive care unit (ICU) patients remain insufficiently characterized. This study investigated the incidence, patterns, burden, and associated factors of severe pDDIs. This single-center retrospective cohort included ICU admissions from January 2023 through December 2024 for adults aged ≥18 years who received ≥2 eligible systemic medications administered orally or intravenously. Category D and X pDDIs were identified with Lexi-Interact (Lexidrug, UpToDate) using confirmed administration records. Logistic and ordinal logistic regression were performed to evaluate severe-pDDI occurrence (≥1) and admission-level burden (0, 1-2, or ≥3). A total of 1,170 ICU admissions were included, involving 26,627 medication administrations. 64.44% were male and 55.38% received mechanical ventilation support. The most common implicated pharmacological group was alimentary-tract and metabolism agents, while ambroxol and glutathione emerged as the top two individual drugs. Among 1,170 ICU admissions, 1,092 (93.33%) exhibited pDDIs, and 769 (65.73%) experienced at least one category D or X pDDI. The 2,704 severe pDDIs comprised 1,681 (62.17%) category D and 1,023 (37.83%) category X events, yielding 231.1 pDDIs per 100 admissions and 101.6 pDDIs per 1,000 medication administrations. Nervous-system agents predominated; butorphanol [948/5,408 ingredient involvements (17.53%)] and dexmedetomidine [824/5,408 (15.24%)] were most frequently implicated. Butorphanol-dexmedetomidine [335/2,704 events (12.39%)] and noscapine-chlorphenamine [176/2,704 (6.51%)] were the leading category D and X pairs, respectively. Additive pharmacodynamic central nervous system depression predominated. Mechanical ventilation and medication count were the strongest adjusted correlates of severe-pDDI occurrence and burden, while baseline variables (sex, APACHE II score) lost significance after adjusting for ICU process variables. These associations remained materially unchanged after excluding 29 readmission encounters. In an exploratory review of 50 severe-pDDI episodes, 32 (64.0%) had temporally associated clinical signs or events documented and 29 (58.0%) had a documented management action. Category D or X pDDI alerts were frequent, primarily driven by pharmacodynamic additive central nervous system depression from sedative-analgesic combinations. Treatment intensity rather than baseline characteristics was associated with severe-pDDI exposure. Regional ICU-tailored surveillance and pharmacist-integrated medication management may facilitate identification of high-risk combinations, although specific drug pairs require validation across different healthcare settings.

PMID 42851402
Read full article →
PubMedActa neurologica Belgica2026-09-27

Ambroxol in Gaucher disease and GBA1-related Parkinson disease: an updated systematic review.

Lipiński Patryk P, Jezela-Stanek Aleksandra A, Tylki-Szymańska Anna A

Ambroxol (ABX) is a central nervous system (CNS)-penetrant compound that can increase glucocerebrosidase activity and has been investigated in Gaucher disease (GD) and GBA1-related Parkinson disease (GBA1-PD). This PRISMA 2020 systematic review and narrative synthesis searched PubMed/MEDLINE, Europe PMC, SSRN and ClinicalTrials.gov through 19 August 2026, with an additional Web of Science Core Collection search performed during revision on 14 September 2026, without restrictions on date, language or publication status. Two reviewers independently selected studies, extracted data and assessed risk of bias using design-specific tools. Substantial clinical and methodological heterogeneity precluded meta-analysis RESULTS: Of 149 records in the original searches, 137 were screened, 41 full texts were assessed and 20 clinical outcome reports were included. The additional Web of Science search retrieved 50 records and did not identify further clinical outcome reports. Evidence in GD consisted mainly of small, uncontrolled and sometimes overlapping reports. Biochemical and neurological improvements were described in selected patients, but outcome definitions and functional responses varied. In GBA1-PD, conclusions remain provisional because the AMBITIOUS results are available only as a non-peer-reviewed preprint and did not show disease modification. Evidence on the safety of chronic high-dose treatment is limited. Biomarker changes alone do not establish clinical efficacy. ABX remains an off-label, investigational treatment in GD and GBA1-PD, and no GBA1 genotype or functional assay currently defines a validated indication.

PMID 42801449
Read full article →
PubMedMolecules (Basel, Switzerland)2026-09-26

Small-Molecule NANT Therapeutics Targeting the Brain-Immune Axis in Alzheimer's Disease: Mechanisms, Clinical Progress, and Translational Challenges.

Sharma Niti N, An Seong Soo A SSA

Alzheimer's disease (AD) is now widely accepted as a complex disorder involving multiple interconnected pathological processes. Increasing evidence suggest that neuroinflammation and immune system dysregulation actively contribute to neurodegeneration, extending beyond the traditional view that the disease is driven solely by amyloid-β (Aβ) and tau pathology. With the advent of studies showing anti-amyloid antibodies to have only modest effects with significant toxicity, there has been a growing interest in investigating non-amyloid non-tau (NANT) treatment approaches. This review provided a general review of small molecules for neuroinflammation-based NANT therapies in AD. We reviewed several agents that affect the brain/immune axis, namely, kinase inhibitors (nelflamaimod and masitinib), NLRP3 inflammasome inhibitors (MCC950, selnoflast, and dapansutrile), TREM2 activators (VG-3927), gingipain inhibitors, PPARγ activators, KCa3.1 inhibitors, and repurposed drugs such as ambroxol and cromolyn. Some common drawbacks were identified for the drugs that failed to provide their anticipated effects. It was found that they all suffered from late-stage treatment, patient heterogeneity, inadequate central nervous system penetration, and poorly designed preclinical studies. Additional translational challenges included sex-specific differences in neuroimmune responses, APOE ε4-associated immune dysfunction, and the need for biomarker-guided patient selection. Although immune-targeted therapies remain strongly supported by biological evidence in AD, meaningful clinical progress will likely require earlier intervention strategies guided by biomarkers and therapies capable of targeting multiple disease pathways simultaneously.

PMID 42796561
Read full article →
PubMedFrontiers in dementia2026-09-22

From engagement to dissemination: a WiseAge patient and public involvement and engagement event on dementia with Lewy bodies.

Aakre Jon Arild JA, Rongve Arvid A, Fredriksen Kari K, Svendsboe Ellen J EJ et al.

Dementia with Lewy bodies may account for up to 20% of dementia cases, yet public awareness remains limited. Public engagement may help communicate dementia research to wider audiences, but little is known about whether information spreads beyond event attendees. This study explored how attendees evaluated information about dementia with Lewy bodies and the ongoing Ambroxol in New and Early Dementia with Lewy Bodies (ANeED) clinical drug trial presented at a WiseAge public engagement event, and how they reported sharing this information afterwards. This descriptive cross-sectional study followed a WiseAge public engagement event that included information about dementia, dementia with Lewy bodies, and the clinical trial. A post-event survey was distributed to attendees, and responses were analysed descriptively. Of 48 attendees, 18 completed the survey. The information was rated positively, with 16 of 18 rating it as good or very good. Attendees reported sharing information beyond the event, most commonly with friends (n = 14) and family members (n = 7), while sharing with neighbors was rare (n = 1). The number of people with whom information was shared ranged from zero to six. No notable change in trial recruitment was observed during the post-event observation period. The event was well received and supported secondary dissemination through attendees' interpersonal networks, particularly friends and family. Although no immediate recruitment effect was observed, public engagement may extend the reach of dementia research communication beyond those who attend.

PMID 42769185
Read full article →
PubMedMetabolic brain disease2026-09-09

Therapeutics candidates and repurposing strategies to target parkinson's disease pathology: current evidence and future directions.

Dhanush Yarava Y, Nizamuddin N D ND, Neelima Satrasala S, Kalpana S S et al.

Parkinson's disease (PD) is the second most prevalent neurodegenerative disorder worldwide, affecting over 8.5 million individuals globally. Its pathophysiology is multifactorial, encompassing progressive loss of dopaminergic neurons in the substantia nigra, accumulation of misfolded alpha-synuclein, mitochondrial dysfunction, oxidative stress, neuroinflammation, and impaired proteostasis. Despite decades of research, current treatments remain predominantly symptomatic, with levodopa and dopamine replacement therapies failing to halt neurodegeneration. Given the high cost and low success rate of de novo drug development, both repurposed approved drugs and mechanistically targeted investigational therapeutics have emerged as promising approaches as a strategically rational alternative that leverages established safety profiles, known pharmacokinetics, and abbreviated regulatory pathways. This review aimed to comprehensively examine the current evidence for repurposed drugs, clinically investigated therapeutic candidates, and repositioning strategies targeting the principal pathological hallmarks of Parkinson's disease: (1) alpha-synuclein aggregation and propagation, (2) oxidative stress, (3) neuroinflammation, (4) mitochondrial dysfunction, (5) lysosomal and proteasomal dysfunction, and (6) dopaminergic neurodegeneration. The review further sought to identify recurring translational challenges, propose mechanistic frameworks for rational combination therapy, and outline future directions for trial design and biomarker integration. A narrative review of published preclinical studies, clinical trials, and recent literature was conducted, focusing on repurposed compounds with mechanistic plausibility and evidence of CNS penetration relevant to PD pathology. Evidence from Phase 1-3 clinical trials and post-mortem neuropathological analyses was synthesised across each pathological hallmark. Compounds were evaluated for mechanistic specificity, pharmacokinetic suitability, and clinical translation status. Across six pathological hallmarks, repurposed agents showed disease-relevant activity, with variable clinical translation. Ambroxol, a GCase pharmacological chaperone, achieved ~ 30-34% brain penetration, increased cerebrospinal fluid alpha-synuclein in a Phase 2 trial (n = 17) regardless of GBA mutation status, and is now in Phase 3 (ASPro-PD, n = 330). N-acetylcysteine restored depleted brain glutathione, scavenged reactive oxygen species, preserved VMAT2 and tyrosine hydroxylase expression, and improved motor outcomes, with positive dopamine transporter imaging in clinical studies. Doxycycline, a BBB-penetrant antibiotic, suppressed microglial MMP-3 and MMP-9 and downregulated TNF-α, IL-1β, iNOS, and COX-2 in preclinical PD models. Metformin and its mitochondria-targeted analog Mito-Met activated AMPK signalling and reversed mitochondrial dysfunction in PD models; however, Mito-Q10 failed in a one-year clinical trial, highlighting that ~ 70% of dopaminergic neurons are already lost at symptom onset, making intervention timing a decisive variable. For proteostatic dysfunction, the USP14 inhibitor IU1 enhanced both proteasomal and autophagic flux, though neurotoxicity above 200 µM limits its therapeutic window; BIIB122, a selective LRRK2 inhibitor, achieved CNS target engagement in Phase 1/2 trials, but its Phase 3 LIGHTHOUSE study was terminated, with Phase 2b LUMA ongoing. In dopaminergic neurodegeneration, GLP-1 receptor agonists produced divergent outcomes: Exenatide-PD3 (n = 231) showed no benefit over placebo, whereas LixiPark demonstrated reduced motor progression in early-stage PD, revealing that mechanistic equivalence does not guarantee clinical equivalence across heterogeneous patient populations. Drug repurposing represents a viable and strategically advantageous approach to targeting PD pathophysiology. Repurposed compounds, including ambroxol, N-acetylcysteine, doxycycline, metformin, and GLP-1 receptor agonists, demonstrate engagement with disease-relevant mechanisms and, in several cases, preliminary evidence of clinical activity. However, a recurring translational challenge is the timing of therapeutic intervention: with approximately 70% of dopaminergic neurons already lost at symptom onset, even mechanistically sound agents may fail if administered too late. Divergent GLP-1 trial outcomes underscore that mechanistic equivalence does not guarantee clinical equivalence, and that disease-stage heterogeneity is a critical confounder. Future priorities must include: (i) precision stratification by genetic profile (e.g., GBA and LRRK2 variants); (ii) combination therapies targeting multiple interconnected hallmarks simultaneously; (iii) development of validated fluid and neuroimaging biomarkers for early-stage patient selection; and (iv) adaptive trial designs that accommodate heterogeneity across the PD population. Integration of computational tools network pharmacology, machine learning, and systems biology with biomarker-defined clinical enrichment will be essential for accelerating repurposed candidates toward meaningful disease modification.

PMID 42714656
Read full article →
PubMedNeuromolecular medicine2026-09-01

Rebalancing α-Synuclein Clearance: Novel Therapeutic Frontiers in Parkinson's Disease.

Jabeen Maryam M, Hameed Huma H, Ahmad Syed Muhammad SM, Mushtaq Tehreem T et al.

Parkinson's disease (PD), the second most common neurodegenerative condition, develops because of abnormal protein misfolding and aggregation of α-synuclein with its subsequent intercellular spread. Such pathological changes lead to disruption of neuronal homeostasis and contribute to neuronal degeneration. During normal conditions, α-synuclein clearance is controlled by different types of lysosomal degradation, namely, macro autophagy, chaperone-mediated autophagy (CMA), micro autophagy, and the ubiquitin-proteasome system. Malfunction of these systems results in increased α-synuclein secretion due to exosome-dependent, direct, and damage-induced mechanisms, which, in turn, promotes enhanced intercellular propagation, inflammation, mitochondrial dysfunction, blood-brain barrier leakage, and neuronal cell death. Although several approaches targeting α-synuclein clearance have shown biological activity in preclinical or early clinical studies, consistent disease-modifying efficacy has not yet been established, owing to challenges including target specificity, blood brain barrier penetration, biological heterogeneity, and the limited sensitivity of clinical endpoints. Recent research indicates that successful treatment is more related to restoring the balance of these two processes than to manipulating one of them.In this review, it is proposed that a systems-level approach can be taken where PD is understood as a disease characterized by the imbalance in proteostasis. Potential treatment modalities include small molecules targeting lysosome function (ambroxol, rapamycin, TFEB activators), CMA enhancers, gene therapy, and antibodies against extracellular α-synuclein. Furthermore, new modalities like molecular glue degraders, allostery-based stabilization of α-synuclein tetramers, engineered decoy particles, and bispecific antibodies represent some other possible routes towards multimodal disease modification.

PMID 42675202
Read full article →

+986 more articles available with a free account

Sign up free to view all articles →

Ask about ambroxol