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Vertex Gets FDA OK to Study VX-407 in Genetic Kidney Disease

By Colin Kellaher


Vertex Pharmaceuticals has won U.S. Food and Drug Administration clearance to study its VX-407 drug candidate in certain patients with the most common inherited kidney disease.

Vertex on Thursday said it will study VX-407 in patients with autosomal dominant polycystic kidney disease, or ADPKD, who have a subset of PKD1 genetic variants.

The Boston drugmaker said it plans to launch a Phase 1 study in healthy volunteers this month, making ADPKD its 10th disease area in the clinic.

Vertex said some 250,000 people in the U.S. and Europe suffer from ADPKD, with no treatments currently available that address the underlying causal biology of the disease. The company added that the majority of ADPKD cases are caused by variants in the PKD1 gene.


Write to Colin Kellaher at


(END) Dow Jones Newswires

March 21, 2024 08:33 ET (12:33 GMT)

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