Pancreatic enzyme prescription following ivacaftor licensing: A retrospective analysis of the US and UK cystic fibrosis registries

Cystic Fibrosis (CF) is a multi-system, autosomal recessive inherited disease caused by mutations to the cystic fibrosis transmembrane conductance regulator (CFTR) gene. CF affects approximately 10,908 people in the UK and 38,804 in the US [1 –3], with an estimated 100,000 people living with CF worldwide [4]. In 2012, the first CFTR potentiator, ivacaftor (VX-770, Kalydeco®, Vertex Pharmaceuticals) was licenced by the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) for those aged 6 years and above with a G551 D mutation in the CFTR gene [5].

Source: Journal of Cystic Fibrosis - February 10, 2024 Category: Respiratory Medicine Authors: Rebecca Calthorpe, Margaret Rosenfeld, Christopher H. Goss, Nicole Green, Mark Derleth, Siobh án B Carr, Alan Smyth, Iain Stewart Tags: Original Article Source Type: research