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FDA Approves Kalydeco (Ivacaftor) for Use in Eight Additional Mutations That Cause Cystic Fibrosis

Disease results from missing or defective CFTR proteins (February 21)

The FDA has approved a supplemental new drug application (sNDA) for Kalydeco (ivacaftor, Vertex Pharmaceuticals) for people aged 6 years and older with cystic fibrosis (CF) who have one of eight additional mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Kalydeco (ivacaftor) was first approved in January 2012 for people aged 6 years and older with CF who have at least one copy of the G551D mutation. With the approval of the sNDA, Kalydeco is now approved for use in people with CF with the following nine mutations: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, and G1349D. In the U.S., approximately 150 people aged 6 years and older have one of the additional eight mutations for which the treatment is now approved.

CF is caused by defective or missing CFTR proteins that result from mutations in the CFTR gene. The defective function or absence of CFTR proteins in people with CF results in poor flow of salt and water into and out of cells in several organs, including the lungs. Ivacaftor facilitates increased chloride transport by potentiating the “channel open” probability (gating) of the CFTR protein.

Kalydeco (ivacaftor) is the first medication to treat the underlying cause of CF in people with specific mutations in the CFTR gene. Known as a CFTR potentiator, the oral treatment aims to help the CFTR protein function more normally once it reaches the cell surface in the lungs, to help hydrate and clear mucus from the airways.

Source: Vertex Pharmaceuticals; February 21, 2014.

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