Acetylcysteine

Evidence Level: L2

Predicted Indications: 10

Acetylcysteine

Pharmacist Assessment Report

Acetylcysteine: Thrombotic disease

One-Sentence Summary

Acetylcysteine is a mucolytic and antioxidant thiol drug that is widely marketed in India as single-ingredient products. The TxGNN model predicts it may be effective for thrombotic disease, with 7 clinical trials and 20 publications compiled for this direction. The strongest support is in thrombotic microangiopathy (TA-TMA) and thrombotic thrombocytopenic purpura (TTP), not thrombosis in general.

Quick Overview

Item	Content
Original Indication	Not recorded in the local data
Predicted New Indication	Thrombotic disease
TxGNN Prediction Score	0.9996
Evidence Level	L2
India Market Status	Listed in a compiled Indian medicine price dataset (at least 20) – CDSCO approval status is not included in this data
Number of Listed Products	at least 20
Recommended Decision	Proceed with Guardrails

Why is This Prediction Reasonable?

Detailed mechanism of action data for the drug is not available in the data compiled for this report. Based on the repurposing analysis, acetylcysteine is a thiol-based reducing agent. It can break disulfide bonds in von Willebrand factor (VWF), reducing multimer size and platelet-binding activity.

This is plausible for VWF-driven microvascular thrombosis such as TTP and transplant-associated thrombotic microangiopathy (TA-TMA). Its antioxidant activity may also reduce oxidative endothelial injury and platelet activation. Preclinical mouse and baboon TTP models support this mechanism (PMID 28011677).

The evidence is strongest for the TMA/TTP subset. It should not be extrapolated to venous or arterial thrombosis in general.

Clinical Trial Evidence

Trial Number	Phase	Status	Enrollment	Key Findings
NCT03252925	Phase 3	Completed	170	Safety and efficacy of N-acetylcysteine in transplant-associated thrombotic microangiopathy; results should be verified
NCT07279610	Phase 2/3	Active, not recruiting	44	Single-arm trial of N-acetylcysteine for TA-TMA; results not yet available
NCT03636932	Phase 2	Completed	40	Randomized, double-blind, placebo-controlled cross-over trial (RENACTIF) on the thrombotic phenotype in renal insufficiency
NCT01808521	Early Phase 1	Completed	3	Pilot of intravenous N-acetylcysteine in suspected TTP; too small for efficacy conclusions
NCT04368598	Phase 2	Unknown	44	Dexamethasone plus acetylcysteine in newly diagnosed immune thrombocytopenia; indirect, combination design
NCT03460808	Phase 1/2	Unknown	200	Atorvastatin, acetylcysteine and danazol in steroid-resistant or relapsed immune thrombocytopenia; indirect, combination
NCT05551624	Early Phase 1	Completed	15	Atorvastatin plus N-acetylcysteine on platelet count in immune thrombocytopenia; indirect, combination

Literature Evidence

PMID	Year	Type	Journal	Key Findings
35940529	2022	Randomized trial	Transplantation and Cellular Therapy	N-acetylcysteine as prophylaxis for TA-TMA in a placebo-controlled study
41977015	2026	Systematic review	Journal of Clinical Medicine	Systematic review and critical appraisal of N-acetylcysteine in TTP
42338865	2026	Systematic review	Cureus	Systematic review of N-acetylcysteine as adjunctive therapy in TTP
37311880	2023	Cohort study	Annals of Hematology	Retrospective study of N-acetylcysteine and in-hospital mortality in acquired TTP
28011677	2017	Preclinical	Blood	N-acetylcysteine in mouse and baboon TTP models
32243196	2020	Review	Expert Review of Hematology	Repurposed drugs for TTP, including N-acetylcysteine
28416507	2017	Review	Blood	Overview of TTP pathophysiology and management
33540569	2021	Review	Journal of Clinical Medicine	TTP pathophysiology, diagnosis and management
36410267	2022	Review	Journal of Infection and Public Health	N-acetylcysteine in COVID-19, which involves a prothrombotic state

India Market Information

Authorization Number	Product Name	Dosage Form	Approved Indication
234623	Vexanac 600 Tablet	strip of 10 tablets	Not recorded
134850	Mucyst 600mg Tablet	strip of 10 tablets	Not recorded
87042	Fluimucil Eff 600mg Tablet	strip of 6 tablets	Not recorded
151932	Nacomac 600mg Effervescent Tablet	strip of 10 effervescent tablets	Not recorded
153551	Nacel 1000mg Injection	vial of 5 ml injection	Not recorded

The data lists 20 valid single-ingredient licences, and the records may be partial. Only the first five are shown. Dosage forms are oral tablets and injectables.

Safety Considerations

Detailed package insert warnings and contraindications are not available in the data compiled for this report; please refer to the package insert.

Drug Interactions: 247 interactions were found in the interaction databases. Listed examples include activated charcoal (minor) and inhaled rapid-acting human insulin (moderate). Many others, such as bupropion, metformin, morphine, omeprazole, prednisone and vancomycin, have an unclassified severity.

Conclusion and Next Steps

Decision: Proceed with Guardrails

Rationale: Evidence level is L2. One completed Phase 3 trial (NCT03252925) and a completed randomized placebo-controlled cross-over trial (NCT03636932) tested acetylcysteine in thrombotic conditions. A randomized TA-TMA prophylaxis trial and systematic reviews in TTP add support. However, there are not multiple completed Phase 3 trials, and the evidence is concentrated in thrombotic microangiopathy rather than thrombosis broadly.

To proceed, the following is needed:

Published results from NCT03252925 and the ongoing NCT07279610
Restrict the claim to TA-TMA/TTP unless broader thrombotic evidence emerges
Package insert warnings and contraindications for safety screening
Mechanism of action data
A review of interactions with anticoagulant and antiplatelet therapy in the target population

Disclaimer

This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.