Drug Overview
VVertex’s Kalydeco is a CFTR potentiator that acts to increase chloride transport through CFTR.
In this way, it mitigates the underlying genetic defect in cystic fibrosis patients with Class III gating mutations, which results in CFTR channels with a reduced ability to transport chloride (Sermet-Gaudelus, 2013).
After initially being approved for cystic fibrosis patients with the G551D mutation, Vertex has subsequently expanded Kalydeco’s label to include patients aged two years and older, now spanning a total of 38 genetic mutations.
VVertex’s Kalydeco is a CFTR potentiator that acts to increase chloride transport through CFTR.
In this way, it mitigates the underlying genetic defect in cystic fibrosis patients with Class III gating mutations, which results in CFTR channels with a reduced ability to transport chloride (Sermet-Gaudelus, 2013).
After initially being approved for cystic fibrosis patients with the G551D mutation, Vertex has subsequently expanded Kalydeco’s label to include patients aged two years and older, now spanning a total of 38 genetic mutations.
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