Vertex gets NDA going for CF triple combo; Disarm woos rare disease expert Alvin Shih as CEO
→ Vertex’s has officially set sail with its latest cystic fibrosis triple combo, filing an NDA to the FDA. Armed with a breakthrough therapy designation, the regimen comprises ivacaftor and tezacaftor — elements of the biotech’s other CF drugs Kalydeco and Symdeko — as well as the novel compound elexacaftor (VX-445). An approval can arrive as soon as March 2020 assuming the priority review request is granted, Jefferies’ Michael Yee notes, adding that the new drug could contribute at least $6 billion in peak sales as it taps into a new population of patients with one F508del mutation and one minimal function mutation.
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