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PF-06743649 is an investigational oral small molecule drug developed by Pfizer as a dual inhibitor of urate transporter 1 (URAT1, encoded by SLC22A12) and xanthine oxidase (XO). Its mechanism of action involves reducing serum uric acid levels through both inhibition of uric acid reabsorption in the kidney (via URAT1) and inhibition of uric acid production (via XO). The drug was primarily developed for the treatment of gout. Clinical studies demonstrated that PF-06743649 could rapidly lower serum uric acid in healthy subjects and patients with gout. However, development was discontinued after phase I trials due to renal safety concerns, specifically cases of acute kidney injury observed during clinical testing[1][2][3][4][7].
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