FDA grants RMAT designation to United Therapeutics’ miroliverELAP®, a bioengineered external liver assist system for acute liver failure (ALF). Bridges unmet needs in temporary hepatic support ahead of transplant. Phase 1 data promising.
Written by: A. Dillikumari, BPharm
Reviewed By: Pharmacally Editorial Team
FDA Grants RMAT Designation for Bioengineered Liver Support System
United Therapeutics Corporation announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to miroliverELAP®, an investigational bioengineered external liver assist system for the treatment of acute liver failure (ALF). The designation is intended to accelerate development of regenerative medicine therapies targeting serious or life-threatening conditions with unmet medical needs.
Acute liver failure is characterized by rapid deterioration of liver function, often progressing within days or weeks. Despite advances in supportive care, treatment options remain limited. Liver transplantation remains the only definitive therapy, yet many patients are either ineligible or unable to receive a donor organ in time. Approximately 45% of patients experience spontaneous recovery, about 25% receive liver transplants, and nearly 30% die due to the rapid disease course and shortage of transplantable organs.
Bioengineered Liver Designed for Temporary Hepatic Support
miroliverELAP®, developed by Miromatrix Medical Inc., a wholly owned subsidiary of United Therapeutics, is designed to provide temporary liver support outside the human body. The system combines an extracorporeal blood circuit with a single-use bioengineered liver intended to sustain patients while awaiting recovery or transplantation.
The bioengineered liver is created using a decellularized porcine liver scaffold seeded with allogeneic human endothelial cells and human liver cells. These cells are derived from donated human livers that are not suitable for transplantation and are supplied by organ procurement organizations. The approach aims to replicate key liver functions and stabilize patients during acute decompensation.
Early Phase 1 Data Support Continued Development
In January, United Therapeutics reported positive results from a Phase 1 study evaluating miroliverELAP in patients with acute liver failure, including acute forms of liver failure, acute-on-chronic liver failure, and severe acute alcoholic hepatitis. Full study results are expected to be published in the second half of 2026. miroliverELAP remains investigational and is not approved for use in any country.
The RMAT designation was supported by preliminary clinical evidence suggesting the therapy’s potential to provide meaningful clinical benefit in this high-risk population. The designation enables increased regulatory interaction, guidance on efficient study design, and potential pathways for accelerated approval, including eligibility for priority review and rolling submission.
Company Highlights Potential to Address Critical Unmet Need
Jeff Ross, Ph.D., President of Miromatrix, stated that the RMAT designation reflects both the significant unmet need in acute liver failure and the promising potential of miroliverELAP as a novel therapeutic option. He added that the company looks forward to continued collaboration with the FDA as development progresses.
Broader Bioengineered Organ Development Pipeline
In addition to miroliverELAP, United Therapeutics and Miromatrix are developing mirokidney®, a fully transplantable bioengineered kidney utilizing the same decellularization and recellularization technology platform. These programs are part of United Therapeutics’ broader organ manufacturing strategy.
The company’s organ and organ-alternative manufacturing efforts span three platforms: xenotransplantation, allogeneic regenerative medicine, and autologous regenerative medicine. These initiatives currently focus on developing bioengineered hearts, kidneys, livers, and lungs, with the goal of addressing the ongoing shortage of transplantable organs for patients with end-stage organ disease.
Reference
United Therapeutics Receives FDA Regenerative Medicine Advanced Therapy Designation for miroliverELAP® for Treatment of Acute Liver Failure, 08 April 2026, United Therapeutics Receives FDA Regenerative Medicine Advanced Therapy Designation for miroliverELAP® for Treatment of Acute Liver Failure – United Therapeutics Investor Relations
About the Writer
DilliKumari is a Pharmacy graduate based in Ariyur, Puducherry. She has a strong interest in pharmacovigilance and is particularly drawn to data analysis within the healthcare field. She is known for her willingness to learn and continuously improve, with a keen attention to detail that sometimes leads her to spend extra time refining her work.