Early Success For Kalydeco Is Just The Beginning For Vertex’s CF Drug
Kalydeco is expected to be a game-changer in cystic fibrosis, but its current label is applicable to only 4% of the patient population. Vertex, however, has an ongoing plan to expand Kalydeco’s label as both a monotherapy and in combination with other experimental CF agents.
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Vertex's cystic fibrosis product Kalydeco is one of five new molecular entities given a positive opinion by Europe's Committee for Medicinal Products for Human Use on May 25, which also included Almirall's novel COPD therapy aclidinium and Novo Nordisk's recombinant Factor XIII.
Interim data from a Phase II study investigating Kalydeco with VX-809 showed statistically significant FEV-1 improvement across dosing groups; the company hopes to start a pivotal Phase III program for the combo in late 2012 or early 2013.
The deputy commissioner detailed the steps involved in getting Vertex’s cystic fibrosis drug rapidly through the approval process and noted that FDA is working on innovative study designs and assessing how to better use diagnostics.