United Therapeutics has announced that the US Food and Drug Administration (FDA) has cleared its investigational new drug (IND) application to initiate a clinical study of the company’s investigational UKidney derived from a 10 gene-edited source pig.
The study will enrol 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.
United Therapeutics expects the first xenotransplant in this trial to be performed around mid-year 2025.
“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, 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 allogeneic kidney transplant.”
This first-in-human clinical study aims to assess safety and efficacy of the UKidney in two groups of participants: ESRD patients who have been assessed and determined to be ineligible for a conventional allogeneic kidney transplant for medical reasons; and ESRD patients who have been on the kidney transplant waitlist but are more likely to die or go untransplanted than receive a deceased donor kidney transplant within five years.
“Eliminating the need for dialysis or limiting time on dialysis may improve survival for many patients with ESRD,” said Noah Byrd, vice president, Global Regulatory Affairs at United Therapeutics. “We appreciate the productive collaboration with the FDA in advancing our efforts to bring this potentially revolutionary therapeutic option to the hundreds of thousands of ESRD patients.”
