Analysts Miffed By Vertex’s Lack Of Transparency With CF Combo Data
This article was originally published in The Pink Sheet Daily
Vertex Pharmaceuticals presented top-line data for its drug combination in cystic fibrosis, but analysts are wondering why the company was not forthcoming about the full data set.
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Despite controversies about incorrectly reported data and allegations of incomplete data, Vertex has demonstrated lung function benefit for a combination of Kalydeco and VX-809 in Phase II and will move the combination into Phase III trials early next year.
FDA’s speedy approval of Vertex’s disease-modifying, genetically targeted cystic fibrosis treatment ivacaftor appears to have been made easier by the company’s failure to show efficacy in a population with a different genetic mutation. The failed study not only had important implications for preventing off-label use, it added considerably to the safety database in an orphan indication.
Kalydeco, an oral CFTR potentiator, becomes the first approved drug to address the underlying cause of cystic fibrosis, rather than just treating the disease’s symptoms.