United Therapeutics receives FDA regenerative medicine designation for liver device

United Therapeutics Corporation (NASDAQ: UTHR) announced that the U.S. Food and Drug Administration granted Regenerative Medicine Advanced Therapy designation to its investigational miroliverELAP, an external liver assist product for treating acute liver failure.

The device, developed by Miromatrix Medical Inc., a wholly owned subsidiary of United Therapeutics, consists of an external blood circuit and a bioengineered liver sustained outside the human body to provide temporary liver support. The bioengineered liver is manufactured by seeding a decellularized porcine liver scaffold with human endothelial cells and human liver cells from donated human livers unsuitable for transplantation.

In January, United Therapeutics reported positive results from its phase 1 study of miroliverELAP in patients with acute forms of liver failure, acute-on-chronic liver failure, and severe acute alcoholic hepatitis. The company plans to publish full study results in the second half of 2026. The device remains investigational and is not approved for any use in any country.

The FDA's RMAT designation accelerates development and regulatory review of regenerative medicine therapies targeting serious or life-threatening diseases with unmet medical needs. The designation provides benefits including intensive FDA guidance, eligibility for priority review, rolling review of Biologics License Application materials, and enhanced organizational commitment from the FDA.

"Receiving this designation from the FDA highlights both the critical unmet need we are addressing and the promising potential of miroliverELAP as a novel therapeutic option," said Jeff Ross, President of Miromatrix.

Acute liver failure affects thousands of patients annually and is characterized by rapid loss of liver function within days or weeks. According to the company, approximately 45% of patients experience spontaneous recovery, 25% receive liver transplants, and 30% die due to ineligibility for transplantation or inability to receive organs in time.

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