Pegrizeprument Granted Orphan Drug Designation for Prevention of Organ Rejection in Patients Receiving a Liver Transplant

CARY, N.C., Jan. 21, 2026 /PRNewswire/ -- Veloxis Pharmaceuticals, Inc. (Veloxis), a global specialty pharmaceutical company part of Asahi Kasei Corporation, announced today that pegrizeprument (also known as VEL-101), a novel investigational maintenance immunosuppressive agent being developed for prevention of acute rejection in solid organ transplant recipients, has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) for prevention of organ rejection in patients receiving a liver transplant. This new designation is a critical step for Veloxis in the development of pegrizeprument and reinforces the unmet need for orthotopic liver transplantation (OLT) patients around the world.

Liver transplantation can be a life-saving treatment for patients with acute liver failure, chronic liver failure, or primary hepatic malignancy. However, OLT patients require life-long immunosuppression, often associated with complex dosing regimens and comorbid toxicities. Despite advancements in post-transplant clinical care, complications of immunosuppression and allograft rejection may negatively impact graft and patient survival. Therefore, a significant unmet need remains for new therapies that address these complications while maintaining adequate immunosuppression.

"We are pleased by the FDA's decision, which highlights the need for groundbreaking therapies to improve outcomes for liver transplant recipients," says Stacy Wheeler, CEO of Veloxis. "This Orphan Drug Designation represents an important step forward for pegrizeprument and reinforces Veloxis' commitment to advancing research and developing innovative therapies that improve the lives of transplant patients."

In the absence of new, novel immunosuppressant options, the Orphan Drug Designation reflects the potential for pegrizeprument to address a critical need for OLT recipients. Veloxis continues to research how pegrizeprument may be able to address unmet needs in the solid organ transplantation population.

Orphan Drug Designation
The FDA Orphan Drug Designation program grants orphan status to investigational drugs and biologics which aim to prevent, diagnose or treat rare diseases and medical conditions that affect fewer than 200,000 people in the United States. This law encourages development of treatments for patients with rare diseases whose conditions are traditionally undertreated. 

About pegrizeprument
Pegrizeprument is a pegylated monoclonal antibody fragment that binds to and blocks CD28-mediated effector-T cell costimulation, without blocking CTLA-4, an important protein found on T cells that naturally helps keep the body's immune responses in check. VEL-101 is, therefore, expected to have a dual mechanism of action where in a direct manner, it blocks CD28-mediated T cell activation, and indirectly, it allows for CTLA-4 mediated immunosuppressive functions. Pegrizeprument is currently being developed for the prevention of acute rejection in solid organ transplant recipients.

Pegrizeprument, also known as VEL-101 and FR104, was licensed by Veloxis Pharmaceuticals, Inc. from OSE Immunotherapeutics in April 2021. As part of the license agreement, Veloxis Pharmaceuticals, Inc. obtained worldwide rights to develop, manufacture, and commercialize pegrizeprument for all transplant indications.

About Veloxis Pharmaceuticals
Veloxis Pharmaceuticals, Inc., an Asahi Kasei company, is a global specialty pharmaceutical company committed to improving the lives of transplant patients. Headquartered in Cary, North Carolina, USA, Veloxis is focused on the global development and commercialization of medications utilized by transplant patients and by patients with serious related diseases. For further information, please visit Veloxis.com.

About Asahi Kasei
The Asahi Kasei Group contributes to life and living for people around the world. Since its foundation in 1922 with ammonia and cellulose fiber business, Asahi Kasei has consistently grown through the proactive transformation of its business portfolio to meet the evolving needs of every age. With more than 50,000 employees worldwide, the company contributes to a sustainable society by providing solutions to the world's challenges through its three business sectors of Healthcare, Homes, and Material. Its Healthcare operations include devices and systems for acute critical care, and manufacture of biotherapeutics, as well as pharmaceuticals. For further information, please visit asahi-kasei.com.

SOURCE Veloxis Pharmaceuticals