21.03.2024 13:45:24 - dpa-AFX: Vertex Gets FDA Nod Of IND For VX-407 For Autosomal Dominant Polycystic Kidney Disease
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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