United Therapeutics has announced FDA clearance of its Investigational New Drug (IND) application for the UKidney xenotransplantation clinical trial. This groundbreaking study marks the first time a clinical trial will assess the potential of a xeno-organ for registration through a Biologics License Application (BLA). The first xenotransplant is anticipated to occur around mid-year 2025.
The trial is designed as a multicenter, open-label, phase 1/2/3 study to evaluate the safety and efficacy of UKidney in patients with end-stage renal disease (ESRD). The study will initially enroll six patients, with the potential to expand to 50 participants. Leigh Peterson, Ph.D., Executive Vice President, Product Development and Xenotransplantation at United Therapeutics, stated, "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."
Trial Design and Endpoints
Participants will undergo a UKidney transplant followed by a 24-week post-transplant follow-up period. After this initial period, participants will be followed for the rest of their lives to monitor survival, UKidney function, and potential zoonotic infections.
Key efficacy endpoints include:
- Participant survival rate
- UKidney survival rate
- Change in measured glomerular filtration rate (GFR)
- Change in quality of life
Safety endpoints include the incidence of adverse events, all-cause mortality, proteinuria, zoonotic infections, and opportunistic infections.
The trial will enroll two groups of ESRD patients: those ineligible for conventional allogeneic kidney transplants and those on the kidney transplant waitlist who are unlikely to receive a deceased donor kidney within five years.
UKidney: A Novel Xenotransplantation Approach
UKidney is an investigational xenokidney derived from a pig with 10 gene edits. Six human genes are added to the pig genome to enhance immunological acceptance and compatibility, while four porcine genes are inactivated to reduce rejection and control organ growth.
Noah Byrd, Ph.D., RAC, Vice President, Global Regulatory Affairs at United Therapeutics, commented, "Eliminating the need for dialysis or limiting time on dialysis may improve survival for many patients with ESRD."
Addressing the Critical Need for Organ Transplantation
According to the American Kidney Fund, over 557,000 patients in the U.S. are on dialysis due to kidney failure. A 2009 study estimated that 52% of kidney transplant candidates over 60 die within five years of being placed on the waitlist. In 2023, only 21,000 deceased donor kidney transplants were performed, highlighting the urgent need for alternative solutions.
United Therapeutics believes that UKidney offers a potential therapeutic alternative to dialysis, addressing the critical shortage of transplantable organs and improving outcomes for patients with ESRD.
