Pegrizeprument Granted Orphan Drug Designation for Prevention of Organ Rejection in Heart Transplant Patients

CARY, N.C., April 22, 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, has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) for the prophylaxis of heart allograft rejection in patients receiving a heart transplant. This follows the recent ODD granted for prevention of organ rejection in patients receiving a liver transplant and represents a significant milestone in Veloxis' mission to address unmet needs in the transplant community.

"We are encouraged by the FDA's decision to grant Orphan Drug Designation for heart transplant patients, which underscores the need for innovative therapies in this population," says Stacy Wheeler, CEO of Veloxis. "This designation for pegrizeprument further demonstrates our commitment to developing groundbreaking therapeutics that improve patient outcomes and quality of life for solid organ transplant recipients."

While orthotopic heart transplantation is a life-saving procedure for patients with heart failure, heart transplant recipients require life-long immunosuppression, which is 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. This designation reinforces Veloxis' commitment to advancing pegrizeprument as a novel, targeted therapy that may address these critical gaps in long-term post-transplant care.

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. Pegrizeprument 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
Asahi Kasei is a diversified global company that contributes to life and living for people around the world. Since its foundation in 1922, with businesses in ammonia and cellulose fiber, Asahi Kasei has consistently grown through proactive portfolio transformation to meet the evolving needs of every age. With 50,000 employees worldwide, the company contributes to sustainability by providing solutions to the world's challenges across its three business sectors: Healthcare, Homes, and Material. For more information, visit https://www.asahi-kasei.com/.

SOURCE Veloxis Pharmaceuticals