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.
Nintedanib is already licensed for treating idiopathic pulmonary fibrosis which is a subtype of ILDs. Nintedanib acts by blocking specific enzymes and pathways that lead to the development of blood vessels within lung cells involved in the scarring process. This inhibits further growth of the scarring tissues resulting in slowing of the disease progression. This action has been shown to be similar in patients with idiopathic pulmonary fibrosis. If licensed, nintedanib may provide the first novel therapy for PF-ILD where limited treatment options currently exist.