Vertex submits CF drug NDA, but no investor Incivek-like excitement
This article was originally published in Scrip
Executive Summary
Vertex Pharmaceuticals took a step closer to being the first company on the US market with a treatment targeting the underlying cause of cystic fibrosis (CF) by submitting its new drug application (NDA) to the US FDA for Kalydeco (ivacaftor), formerly known as VX-770, an oral drug that targets the defective protein that causes CF, known as CF transmembrane conductance regulator (CFTR), a ATP-binding cassette transporter protein.