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Modulator therapies, manufactured by Vertex Pharmaceuticals (VRTX.O), may no longer be available for new cystic fibrosis (CF) patients in the UK. A draft guidance recently published by the National Institute for Health and Care Excellence (NICE) stated that it would not recommend the use of Kaftrio, Symkevi and Orkambi on new CF patients. Kaftrio and Symkevi are currently recommended for the treatment of cystic fibrosis in patients aged 6 years and older, while Orkambi is used for patients aged 1 and older. The NICE draft guidance highlighted medicine costs and the strain on resources and finances faced by the National Health Service (NHS) as reasons for removing these drugs.

PBR Staff Writer Published 24 February 2014 US-based biotechnology firm Vertex Pharmaceuticals has received approval from the US Food and Drug Administration (FDA) for a supplemental New Drug Application (sNDA) of its Kalydeco (ivacaftor) for the treatment of people with cystic fibrosis (CF).