The US FDA has granted a supplemental new drug application (sNDA) for Vertex Pharmaceuticals’ lead cystic fibrosis treatment Kalydeco (ivacaftor) to treat CF patients aged 6 years and older who carry the R117H mutation in the CF transmembrane conductance regulator (CFTR) gene.
An estimated 500 people or more in the US have the R117H mutation. With Kalydeco’s sNDA, over 3,100 people in North America, Europe and Australia will have a new treatment option.
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