Introduction: The unmet need and the growing prevalence of hyperuricemia and its complications worldwide have pushed investigators to identify new agents to manage hyperuricemia.
Areas covered: This review discusses the drugs in preclinical and early clinical trials for hyperuricemia, their mechanisms of action and available results. This article reviews a total of 10 novel agents: i) drugs in Phase II/III trials - arhalofenate (MBX201), AC201, RDEA group of drugs (including lesinurad), tranilast, ulodesine (BCX4208); and ii) drugs in Phase I trials, including levotofisopam, UR1102, KX1151, LC350189 and Marine Active.
Expert opinion: The goal of emerging therapies is to address the unsatisfactory control of serum uric acid in patients with symptomatic hyperuricemia such as those with gout, to provide better tolerability compared to traditional agents and minimize the risk of adverse events, especially in patients with comorbidities and the elderly. Some drugs like arhalofenate, ulodesine (BCX-4208) and lesinurad are in or have completed Phase II and Phase III trials. The growing knowledge about the urate transporters in the kidney have advanced our knowledge of pathophysiology of hyperuricemia and have led to the development of several new potential treatment options. Availability of new drugs will lead to better management and address the unmet need in patients with symptomatic hyperuricemia in the coming years.
Keywords: AC-201; KX1151; LC350189; Marine Active; OAT4; RDEA; UR1102; arhalofenate; lesinurad; levotofisopam; purine nucleoside phosphorylase; tranilast; ulodesine (BCX-4208); urate anion transporter 1.