CAMBRIDGE (MASSACHUSETTS) (dpa-AFX) - Thursday, Vertex Pharmaceuticals Inc. (VRTX) announced that the FDA has cleared the Investigational New Drug Application or IND for VX-407 for the treatment of autosomal dominant polycystic kidney disease or ADPKD.
VX-407 is a small molecule corrector that targets the root cause of ADPKD, a genetic kidney disease that shortens life expectancy.
ADPKD affects patients with specific PKD1 genetic variations and is characterized by the development of multiple kidney-enlarging cysts. These cysts impair kidney function and can eventually result in kidney failure, necessitating dialysis or a kidney transplant, and causing premature death.
The company is also planning to initiate a Phase 1 clinical trial in healthy volunteers this month.
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