In short
Erdosteine is a mucolytic medicine, a type of drug that helps break down thick mucus in the airways. It has an orphan designation in the European Union for the treatment of lead toxicity, and its main studied use is in chronic obstructive pulmonary disease (COPD). One authorised Phase 3 trial (NCT07329946) is investigating its effect on oxidative stress and inflammation in 30 patients with COPD.
Key points
- Erdosteine is a mucolytic substance that helps break down thick mucus.
- It holds an orphan designation in the EU for the treatment of lead toxicity.
- One Phase 3 trial is studying erdosteine in COPD.
- The trial involves 30 participants and is currently authorised.
- The main measure is the change in glutathione peroxidase 2 (GPX2) expression in blood.
- Erdosteine is given as 600 mg capsules by mouth.
What is erdosteine?
Erdosteine is a mucolytic substance, meaning it helps break down thick mucus in the airways so it can be coughed up more easily. It is also known as dithiosteine and has the chemical formula C8H11NO4S2 with a molecular weight of 249.30. In the European Union, erdosteine has been granted an orphan designation for the treatment of lead toxicity, a rare condition caused by exposure to lead. The designation was granted on 6 December 2012 to Rafifarm SRL, Romania. Erdosteine is already used as a medicine in some countries, where it is marketed for respiratory conditions.
How does erdosteine work?
Erdosteine works by breaking down the structure of mucus in the airways. Its two thiol groups are blocked within the molecular structure, which means it does not directly affect gastric mucus. In the ongoing COPD trial, researchers are investigating whether erdosteine can also influence oxidative stress, inflammatory response and immune modulation. The main focus is on the expression of glutathione peroxidase 2 (GPX2), an enzyme involved in protecting cells from oxidative damage.
Which conditions is erdosteine used or studied for?
- Chronic obstructive pulmonary disease (COPD) — a long-term lung condition that makes breathing difficult. The authorised Phase 3 trial is testing whether erdosteine can reduce oxidative stress and inflammation in people with COPD.
- Lead toxicity — a rare condition caused by exposure to lead, which can damage the heart, kidneys, bones, nervous system and other organs. Erdosteine has an orphan designation in the EU for this condition.
How is erdosteine given?
Erdosteine is given by mouth as capsules. The trial in COPD uses ESTECLIN 300 mg capsules, with a total dose of 600 mg taken orally. DrugCentral lists a dose of 0.60 g by oral route.
What do the clinical trials show?
One clinical trial is listed in the registries for erdosteine. It is a Phase 3, single-centre, open-label, double-arm, controlled study lasting 4 weeks. The trial is currently authorised, meaning it has been approved but may not yet have started recruiting. It plans to enrol 30 participants with COPD. The study is explorative and ex vivo, meaning samples are taken from patients and analysed outside the body. The primary outcome is the difference from baseline in relative GPX2 expression and GPX2 enzyme quantification in peripheral blood after 30 days of treatment with erdosteine plus standard of care.
What side effects are known?
Erdosteine has a good safety profile. The incidence of gastrointestinal side effects such as nausea, heartburn and stomach pain is approximately 7%, which is no higher than placebo. Taste disturbances, including ageusia and dysgeusia, have been reported occasionally. Unlike some other thiol-containing compounds, erdosteine does not cause gastric reflux or an unpleasant smell. No evidence of interaction between erdosteine and other drugs, including antibiotics, theophylline and beta-2 agonists, has been found.