Cysteamine bitartrate for cystic fibrosis exacerbations

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Indications: Cystic fibrosis
Therapeutic Areas: Genetic Disorders , Respiratory System
Year: 2018

Oral cysteamine bitartrate (capsule) is in clinical development to treat pulmonary exacerbations in people with cystic fibrosis (CF). CF is a life-limiting, inherited disease caused by a defective gene which results in lungs, the digestive system, and other tissues becoming congested with thick mucus. The presence of the mucus in the airways of CF patients leads to numerous health issues, including chronic and recurrent infections of the lungs and consequent inflammation, reduction of lung function, and breathing difficulties. Some patients with CF may be symptom-free when well, but many will have chest symptoms such as cough and sputum production.
Cysteamine bitartrate as an oral formulation is a novel treatment of exacerbations of CF-associated lung disease, with a unique multi-action: breaking down of the excessive mucus produced by the lining of the airways, killing of the bacteria responsible for the recurrent respiratory infections, and disrupting the biofilms in which they colonise. Cysteamine bitartrate also potentiates the activity of standard of care antibiotics used in CF patients, and is potentially antibiotic-sparing as an adjunct treatment alongside standard of care therapy. If licensed, cysteamine bitartrate will offer an additional treatment option for adult patients with CF exacerbations.