New drugs, new challenges in cystic fibrosis care.

Cystic fibrosis (CF) is a genetic disease caused by variants in the gene encoding for the CF transmembrane conductance regulator (CFTR) protein, a chloride and bicarbonate channel. CFTR dysfunction results in a multiorgan disease with the main clinical features being exocrine pancreatic insufficiency and diffuse bronchiectasis with chronic airway infection leading to respiratory failure and premature death. Over the past decades, major progress has been made by implementing multidisciplinary care, including nutritional support, airway clearance techniques and antibiotics in specialised CF centres. The past decade has further seen the progressive development of oral medications, called CFTR modulators, for which around 80% of people with CF are genetically eligible in Europe. CFTR modulators partially restore ion transport and lead to a rapid and major improvement in clinical manifestations and lung function, presumably resulting in longer survival. CFTR modulators have been game-changing in the care of people with CF. However, many questions remain unanswered, such as the long-term effects of CFTR modulators, especially when treatment is started very early in life, or the new CF-related disease emerging due to CFTR modulators. Moreover, severe complications of CF, such as diabetes or cirrhosis, are not reversed on CFTR modulators and around 20% of people with CF bear

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Conflict of interest: I. Fajac reports grants from AbbVie, Bayer, Boehringer Ingelheim, Insmed, GSK, Vertex Pharmaceuticals and Zambon; consulting fees from AbbVie, Boehringer Ingelheim, Genvade, Kither Biotech and Vertex Pharmaceuticals; lecture honoraria from Vertex Pharmaceuticals; and a leadership role as President of the European Cystic Fibrosis Society, outside the submitted work. P-R. Burgel reports grants from Vertex Pharmaceuticals and GSK; consulting fees from AstraZeneca, Chiesi, GSK, Insmed, Vertex, Viatris and Zambon; and travel support from AstraZeneca and Chiesi, outside the submitted work. C. Martin reports lecture honoraria from AstraZeneca, Chiesi and Zambon; travel support from Chiesi, Sanofi and Zambon; and advisory board participation with Vertex, Zambon and GSK, outside the submitted work.