United Therapeutics (NASDAQ:UTHR), a leader in regenerative medicine and organ manufacturing, announced on April 8, 2026, that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to its investigational miroliverELAP®.
The miroliverELAP system is a first-of-its-kind external liver assist product designed to provide temporary support for patients suffering from acute liver failure (ALF), including acute-on-chronic liver failure and severe acute alcoholic hepatitis.
Developed by UTHR’s wholly owned subsidiary, Miromatrix Medical, the technology utilizes a bioengineered liver—created by seeding a decellularized porcine scaffold with human cells—to function as an external blood circuit.
The RMAT designation is a significant regulatory milestone, reserved for innovative therapies that demonstrate preliminary clinical evidence of the potential to address life-threatening conditions.
This status provides United Therapeutics with intensive FDA guidance on study design, eligibility for priority and rolling reviews, and an expedited pathway toward potential Biologics License Application (BLA) approval.
The bioengineered liver at the heart of the system is manufactured using allogeneic human endothelial and liver cells isolated from donated organs that were deemed unsuitable for traditional transplantation.
This approach effectively creates a functional "auxiliary" organ that can perform essential metabolic tasks outside the patient's body.
The announcement follows positive Phase 1 clinical results reported in January, which demonstrated the system's safety and preliminary utility in ALF patients.
United Therapeutics confirmed that full study results are slated for publication in the second half of 2026.