United Therapeutics Corporation announced FDA clearance to initiate its landmark UHeart Xenotransplantation Clinical Trial. The regulatory agency cleared the Investigational New Drug application for a novel porcine-derived heart. This investigational organ, known as UHeart, comes from a pig engineered with 10 specific gene edits. Named EXPRESS, the study represents the first human clinical trial of a xenoheart aiming for potential regulatory registration. The company ultimately plans to submit a Biologics License Application using data compiled from this research program.
Kristina DeSmet, Ph.D., DABT, highlighted the scientific momentum behind this medical milestone.
“Moving a porcine-derived heart into human clinical trials represents another defining advancement for the field of xenotransplantation.”
The Senior Director, Product Development at United Therapeutics, also described how this trial builds upon the surgical history of the company.
“The heart is one of the most complex solid organs to transplant, and proceeding into the clinic reflects years of coordinated scientific progress. For United Therapeutics, this milestone represents our third clinical trial in xenotransplantation and underscores the breadth of our platform, spanning end-stage renal disease and now life-threatening heart disease. Together, these programs reinforce our commitment to expanding transplant options for patients who currently have no other alternatives.”
Noah Byrd, Ph.D., RAC, Vice President, Global Regulatory Affairs at United Therapeutics, commented on the current options available to critical cardiac patients.
“Patients with end-stage heart disease continue to face profound limitations in available treatment options. This FDA clearance to proceed with our EXPRESS clinical trial will allow us to begin evaluating an innovative therapeutic option designed to address this unmet need.”
Structure and Criteria of the EXPRESS Trial
The EXPRESS trial operates as a single-center, seamless phase 1/2/3 study. This unique structure allows investigators to evaluate safety and efficacy without moving through completely separate, traditional clinical phases. Initially, the trial will enroll up to two participants. The company will share data with the FDA after tracking the first recipient for 12 weeks. Approval from the regulatory agency is required before performing the second surgery.
Candidates must meet strict criteria to qualify for the surgical study. Individuals must be 50 years of age or older. Additionally, they must have advanced heart failure classified as American College of Cardiology/American Heart Association stage D and New York Heart Association Class IV. Eligible patients must possess no other therapeutic options.
Medical teams will utilize a crossmatch assay to confirm immunological compatibility. Patients are excluded if they need multiple organ transplants or have a history of solid organ transplants. Individuals requiring venoarterial ECMO support cannot participate. Furthermore, candidates must not have severe co-morbidities like chronic liver disease, severe central vascular disease, severe neurologic diseases, or uncontrolled diabetes. A history of medical noncompliance also disqualifies an individual.
The primary protocol enforces a 24-week post-transplant follow-up period to evaluate function and exercise capacity. After this stage, the patients will be monitored throughout their lives for any signs of possible zoonotic infection. There will be an independent Data Monitoring Committee that will consider safety results. They will monitor the occurrence of arrhythmia, stroke, and opportunistic infections.
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News Source: Businesswire.com
