Press release
United Therapeutics Corporation Announces FDA Clearance of its Investigational New Drug Application for the UKidney Xenotransplantation Clinical Trial
First-ever human clinical trial of a xeno-organ intended to allow for potential registration through the submission of a Biologics License Application to the
About this update from United Therapeutics Corporation
[{"type":"text","content":"\nFirst-ever human clinical trial of a xeno-organ intended to allow for potential registration through the submission of a Biologics License Application to the U.S. FDA\n\n\nFirst xenotransplant is expected to occur around mid-year 2025\n\n\n SILVER SPRING, Md. & RESEARCH TRIANGLE PARK, N.C.--(BUSINESS WIRE)--\nUnited Therapeutics Corporation (Nasdaq: UTHR), a public benefit corporation, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug application (IND) to initiate a clinical study of the company’s investigational UKidney™ derived from a 10 gene-edited source pig. The study will enroll an initial cohort of six end-stage renal disease (ESRD) patients, expanding to up to 50 participants, and is intended to support a Biologics License Application (BLA) with the FDA.\n\n\nUnited Therapeutics expects the first xenotransplant in this trial to be performed around mid-year 2025.\n\n\nAccording to the American Kidney Fund, more than 557,000 patients in the U.S. are on dialysis to filter their blood when their kidneys are no longer able to do so1. A 2009 study estimated that 52% of kidney transplant candidates who were at least 60 years of age when placed on the transplant waitlist die within five years before receiving a transplant2. United Therapeutics believes that xenotransplantation offers a therapeutic alternative to dialysis.\n\n\n“Clearance of our IND for this first-ever clinical trial of a xenokidney represents a significant step forward in our relentless mission to expand the availability of transplantable organs,” said Leigh Peterson, Ph.D., Executive Vice President, Product Development and Xenotransplantation at United Therapeutics. “Our goal is to increase the availability of transplantable organs to offer a therapeutic alternative to a lifetime on dialysis for a large population of patients who are unlikely to receive an allogeneic3 kidney transplant.”\n\n\nThis first-in-human clinical study aims to assess safety and efficacy of the UKidney in two groups of participants:\n\n\n\nESRD patients who have been assessed and determined to be ineligible for a conventional allogeneic kidney transplant for medical reasons; and\n\n\n\nESRD patients who have been on the kidney transplant waitlist but are more likely to die or go untransplanted than receive a deceased donor kidney ...