Nintedanib is an oral medicinal product under clinical development for patients with progressive fibrosing interstitial lung disease (PF-ILD). ILDs belong to a group of over 200 rare conditions that affect the tissue and space around the air sacs of the lungs, where tissue becomes thickened, stiff and scarred over a period of time. PF-ILDs are a specific sub-type where there is a progressive scarring (fibrosis) of the lung tissue that results in worsening respiratory symptoms, lung function decline, limited response to immunomodulatory therapies, organ failure, decreased quality of life and, potentially, early death.
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.
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.