Influence of Decisions Related to Business and Regulatory Considerations on Therapeutic Development for Kidney Diseases

[摘要] Much has been written about the growing prevalence of CKD, especially CKD caused by diabetic nephropathy. Despite increased awareness of CKD as a worldwide medical problem, there remain few pharmacological therapies available to prevent or slow the progression of CKD to ESRD, much less therapies that result in a cure. The only drugs approved by the Food and Drug Administration (FDA) for CKD (specifically diabetic nephropathy) are drugs that inhibit angiotensin-converting enzyme (ACEi) and angiotensin receptor blockers (ARBs). Captopril (ACEi) is approved for use in type I diabetic patients with nephropathy and retinopathy (1), and losartan and irbesartan (ARBs) are approved for the treatment of type II diabetic patients with nephropathy, elevated serum creatinine, proteinuria, and hypertension (2,3). None of these drugs were initially developed for the treatment of CKD in general or diabetic nephropathy specifically. Although these classes of drugs have been major breakthroughs, clearly slow the progression of diabetic nephropathy (4–6), and are routinely used in the treatment of other forms of CKD, they do not stop the progression of CKD. To my knowledge, the FDA has approved no drugs with the initial indication being cure or slowing of the progression of CKD. Thus, in addition to extant therapies, new pharmacological therapies for CKD are needed. Why, then, are more novel drugs not available? Although I do not have the answers, some thoughts about this question will be provided based on a career spent mostly in academic medicine and the pharmaceutical industry. Review of specific potential targets or drug candidates in development for CKD is beyond the scope of this discussion. Although not mentioned, there is also a pressing need for new therapies for acute kidney injury (AKI).

[学科分类] 泌尿医学

[关键词] Bone marrow necrosis;Sickle cell disease;Hyperhemolysis syndrome