United Therapeutics Corporation Receives U.S. Food and Drug Administration Regenerative Medicine Advanced Therapy (RMAT) Designation for miroliverELAP® in the Treatment of Acute Liver Failure
United Therapeutics Corporation (Nasdaq: UTHR), a public benefit corporation focused on developing innovative therapies for end-stage organ diseases, has announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to its investigational product miroliverELAP®, an external liver assist system designed to support patients with acute liver failure (ALF). The designation represents an important regulatory milestone for the program and underscores both the severity of the condition being addressed and the potential of the therapy to meet a significant unmet medical need.
miroliverELAP was developed by Miromatrix Medical Inc., a wholly owned subsidiary of United Therapeutics that specializes in organ manufacturing technologies. The therapy consists of an external blood circuit connected to a single-use bioengineered liver that is maintained outside the human body. This external liver is designed to temporarily perform essential liver functions in patients experiencing acute liver failure, providing critical metabolic and detoxification support while the patient’s own liver attempts to recover or while awaiting transplantation.
The bioengineered liver component of miroliverELAP is created using a proprietary process that involves decellularization and recellularization of donor organ scaffolds. Specifically, the platform utilizes decellularized porcine liver scaffolds that have been stripped of their cellular components while preserving the underlying extracellular matrix structure. These scaffolds are then seeded with allogeneic human endothelial cells and hepatocytes derived from donated human livers that are not suitable for transplantation. These donor organs are typically provided through organ procurement organizations and would otherwise not be used for transplant purposes.
By combining advances in tissue engineering, cell biology, and biomanufacturing, the miroliverELAP system aims to replicate key functions of a native human liver in an extracorporeal setting. The external device is intended to act as a temporary bridge therapy, supporting patients during acute episodes of liver failure when the organ is unable to sustain vital physiological processes.
The RMAT designation granted by the FDA is part of a specialized regulatory program designed to accelerate the development and review of regenerative medicine therapies targeting serious or life-threatening diseases. To qualify for RMAT designation, a therapy must demonstrate preliminary clinical evidence indicating that it has the potential to treat, modify, reverse, or cure a serious condition with significant unmet medical need. The designation is similar in intent to other expedited programs such as Breakthrough Therapy designation but is specifically tailored to regenerative medicine products, including cell therapies, tissue-engineered products, and certain combination therapies.
With RMAT designation, sponsors benefit from enhanced and more frequent interactions with the FDA throughout the development lifecycle. These interactions include guidance on clinical trial design, selection of endpoints, and strategies for demonstrating safety and efficacy. In addition, RMAT-designated therapies may be eligible for priority review, rolling submission of Biologics License Application (BLA) components, and potential use of surrogate or intermediate clinical endpoints to support accelerated approval pathways. Collectively, these advantages are intended to streamline development timelines while maintaining rigorous regulatory standards.
Jeff Ross, Ph.D., President of Miromatrix, commented on the significance of the designation, noting that it reflects both the urgent unmet need in acute liver failure and the promising potential of miroliverELAP as a novel therapeutic option. He emphasized the company’s commitment to continuing collaborative engagement with the FDA as the program advances through clinical development. According to Ross, the designation represents an important validation of the underlying technology and its potential to address a critical gap in current treatment options.
Acute liver failure is a rapidly progressing and often fatal condition characterized by the sudden loss of liver function in patients without pre-existing chronic liver disease. The condition can develop over a period of days or weeks and is associated with high mortality rates if not managed promptly. Causes of ALF can include drug-induced liver injury, viral hepatitis, autoimmune disorders, and acute alcoholic hepatitis, among others.
Current treatment options for ALF are limited. In some cases, patients may experience spontaneous recovery, but many require intensive supportive care in a hospital setting. Liver transplantation remains the only definitive treatment for patients with irreversible liver failure. However, the availability of suitable donor organs is severely limited, and the rapid progression of the disease often makes timely transplantation challenging. As a result, a significant proportion of patients either do not qualify for transplant or are unable to receive one in time, contributing to high mortality rates associated with the condition.
United Therapeutics and Miromatrix are developing miroliverELAP with the goal of providing a bridge therapy that can stabilize patients during acute episodes of liver failure. By temporarily replicating key liver functions outside the body, the system is designed to support patient survival and potentially allow time for native liver recovery or access to transplantation. In January, the company reported positive results from a Phase 1 clinical study evaluating miroliverELAP in patients with acute forms of liver failure, acute-on-chronic liver failure, and severe acute alcoholic hepatitis. These indications are collectively categorized under the broader umbrella of acute liver failure (ALF).
The Phase 1 study results provided initial clinical evidence supporting the safety and feasibility of the miroliverELAP system in a real-world patient population. The full dataset from this study is expected to be published in the second half of 2026, which will provide additional insights into the therapy’s performance, including potential signals of efficacy and durability of support. While the product remains investigational and is not approved for any use in any jurisdiction, these early results are an important step in its clinical development pathway.
In addition to miroliverELAP, United Therapeutics and Miromatrix are applying similar organ engineering technologies to the development of other bioengineered organs. One such program is mirokidney®, a fully transplantable bioengineered kidney designed using the same decellularization and recellularization approach employed in miroliverELAP. These efforts are part of a broader strategy to address the global shortage of transplantable organs through innovative manufacturing platforms that combine elements of xenotransplantation, allogeneic regenerative medicine, and autologous regenerative medicine.
United Therapeutics’ organ manufacturing initiatives span multiple organ systems, including hearts, kidneys, livers, and lungs. The company’s long-term objective is to create scalable, transplantable, and functionally viable organs that can reduce reliance on donor organ availability and improve outcomes for patients with end-stage organ disease. By investing in multiple technological platforms, the company aims to build a comprehensive portfolio of organ alternatives capable of addressing critical gaps in transplantation medicine.
The granting of RMAT designation for miroliverELAP represents a meaningful advancement in this broader mission. It not only facilitates closer regulatory collaboration but also reinforces the potential of regenerative medicine approaches to transform the treatment landscape for life-threatening conditions such as acute liver failure. As the program progresses through clinical development, further data will be essential to determine the safety, efficacy, and scalability of this novel external liver assist system and its potential role in clinical practice.
Overall, the FDA’s RMAT designation highlights both the promise of miroliverELAP and the growing importance of regenerative medicine technologies in addressing some of the most challenging unmet needs in healthcare.
About United Therapeutics
Founded by CEO Martine Rothblatt to discover a cure for her daughter’s life-threatening rare disease, pulmonary arterial hypertension, United Therapeutics transforms the treatment of rare diseases and pioneers alternatives to expand the supply of transplantable organs. From our innovative therapies to our groundbreaking manufactured organs, we are bold and unconventional. We move quickly from scientific theory to practical technologies that can save lives. As a public benefit corporation, even our legal structure reflects our commitments. We serve patients, act with integrity, create long-term shareholder value, and operate with sustainable practices that protect the future we are working to build.
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