FDA grants orphan drug designation to NephRx's lead product NX001

NephRx Corporation today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its lead product NX001 for the prevention of delayed graft function (DGF) in renal transplant recipients. NX001 is a kidney growth factor peptide that has demonstrated good safety and promising efficacy in two animal models of acute renal failure.  

"Delayed graft function is a common condition affecting many kidney transplant patients, but current treatment options are limited," said James Koziarz, Ph.D., CEO of NephRx Corp.  "NX001 has demonstrated promising activity in improving kidney function in preclinical models of acute renal failure, and we look forward to initiation later this year of our clinical program to test its effectiveness in helping patients achieve better kidney function post-transplant."

DGF affects an estimated 20-25% of the approximately 14,000 renal transplants performed annually in the U.S.  Currently, there is no treatment for DGF.  DGF patients are put on dialysis to "rest" the transplanted kidney, correct metabolic imbalances and wait for kidney function to return spontaneously.  Studies have shown that DGF can negatively affect future graft function and can increase the incidence of acute rejection episodes.  Kidney grafts with impaired function require the most intense follow-up and therapeutic management and are economically most costly.  

"NX001 is an entirely new approach to treating acute kidney dysfunction," noted F. Gary Toback, M.D., Ph.D., Founding Scientist of NephRx and Professor of Medicine and Cell Physiology at the University of Chicago Medical School.  "The kidney is one of the few human organs with the potential for self-repair, and we believe a kidney growth factor peptide such as NX001 has the potential to stimulate improved renal functioning in a number of renal disorders.  DGF represents a well-defined condition where we hope to show that NX001 can improve near-term kidney function in Phase II trials planned for mid-2011."

Orphan drug is a designation by the U.S. FDA under the Orphan Drug Act confirming that a new drug in development is intended to treat a rare disease, defined as those that affect fewer than 200,000 persons in the United States.  Sponsors of drugs granted orphan designation qualify for tax credits and marketing exclusivity incentives.