The US Food and Drug Administration approved a new cystic fibrosis transmembrane conductance regulator (CFTR) modulator treatment for use in patients ≥12 years of age with 2 copies of the most common CF mutation, F508del.

Tezacaftor/ivacaftor (Symdeko™; Vertex Pharmaceuticals) can also be used in patients with a copy of ≥1 of the other 26 specified mutations of the key protein involved in CF, including E56K, R117C, A455E, P67L, and E193K.

The drug combination improved lung function by 4 percentage points in patients with 2 copies of the F508del mutation compared with placebo in late-stage clinical trials. 

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Participants in these trials also experienced a 35% reduction in exacerbations. In participants with a copy of ≥1 of the other 26 specified mutations, lung function improved by 6.8 percentage points.

Researchers also compared this CFTR treatment with lumacaftor/ivacaftor and found that patients who were given tezacaftor/ivacaftor were less likely to experience chest tightness and potential drug interactions.

The Cystic Fibrosis Foundation offered clinical, scientific, and funding support for the development of tezacaftor/ivacaftor.

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FDA approves new CFTR modulator treatment for cystic fibrosis [press release]. Bethesda, MD: Cystic Fibrosis Foundation; February 12, 2018. Accessed February 13, 2018.