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OCTOBER 21, 2019

FDA Approves Trikafta for Cystic Fibrosis

By SPC News Staff

The FDA approved elexacaftor-ivacaftor-tezacaftor (Trikafta, Vertex Pharmaceuticals), the first triple-combination therapy available to treat patients with the most common cystic fibrosis (CF) mutation. 

Elexacaftor-ivacaftor-tezacaftor is approved for patients aged 12 years and older with CF who have at least one F508del mutation in the CF transmembrane conductance regulator (CFTR) gene, which is estimated to represent 90% of the CF population. The presence of at least one