New Hope for Liver Transplants: Veloxis Drug Earns Key FDA Status

CARY, N.C. – January 21, 2026 – For the thousands of patients who receive a life-saving liver transplant each year, the surgery is only the beginning of a lifelong medical journey. This journey is dominated by a strict regimen of immunosuppressive drugs designed to prevent the body from rejecting the new organ, but these powerful medications often come at a cost, carrying a heavy burden of toxic side effects. Now, a new beacon of hope has emerged on the horizon.

Veloxis Pharmaceuticals, a Cary-based specialty pharmaceutical company, announced today that its investigational drug, pegrizeprument, has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA). The designation is specifically for the prevention of organ rejection in patients receiving a liver transplant, a move that validates the significant unmet need for safer, more effective post-transplant therapies.

The Lifelong Burden of Immunosuppression

Liver transplantation is a modern medical miracle, offering a second chance at life for individuals with acute liver failure, chronic liver disease, or liver cancer. However, the immune system is hardwired to identify and attack foreign tissue, including a transplanted organ. To counteract this, patients must take a cocktail of immunosuppressant drugs for the rest of their lives.

While these medications are effective at preventing rejection, their long-term use is a delicate balancing act fraught with complications. The current standard of care often involves calcineurin inhibitors like tacrolimus and cyclosporine, which are known for their potential to cause severe kidney damage, high blood pressure, and tremors. Other common agents include steroids, which can lead to weight gain, bone thinning, and new-onset diabetes, and antimetabolites like mycophenolate mofetil, which can cause gastrointestinal distress and lower white blood cell counts, increasing the risk of serious infections.

This complex web of side effects can dramatically impact a patient's quality of life and may ultimately threaten the long-term survival of both the patient and the transplanted organ. The medical community has long sought a new generation of therapies that can maintain adequate immunosuppression without the associated toxicities, a challenge that pegrizeprument aims to address.

A Novel Approach to Preventing Rejection

Pegrizeprument, also known as VEL-101, represents a more targeted approach to modulating the immune system. It is a pegylated monoclonal antibody fragment engineered to selectively block a specific pathway involved in activating the immune response. The drug works by binding to and blocking CD28, a protein on T-cells that provides a critical “go” signal for an immune attack.

Crucially, pegrizeprument is designed not to block CTLA-4, another important protein on T-cells that acts as a natural brake on the immune system. This dual mechanism is a key differentiator. By directly blocking the T-cell activation signal (CD28) while indirectly allowing the body's own immunosuppressive brake (CTLA-4) to function, the drug aims to downregulate the aggressive effector T-cells responsible for rejection while potentially promoting regulatory T-cells that foster tolerance. This precision could lead to effective rejection prevention with a more favorable safety profile compared to the broad-spectrum immunosuppressants currently in use.

“We are pleased by the FDA's decision, which highlights the need for groundbreaking therapies to improve outcomes for liver transplant recipients,” said Stacy Wheeler, CEO of Veloxis, in a company statement. “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.”

The Strategic Power of Orphan Drug Designation

The FDA's Orphan Drug Designation is more than just a regulatory milestone; it is a powerful strategic tool designed to accelerate the development of treatments for rare diseases. A condition is considered rare if it affects fewer than 200,000 people in the United States. While thousands of liver transplants are performed annually, the number falls within this threshold, making pegrizeprument eligible for the program.

This designation provides Veloxis with a suite of significant incentives that help de-risk the costly and lengthy process of drug development. Most importantly, upon potential FDA approval, the company will be granted seven years of market exclusivity for pegrizeprument in the liver transplant indication. This protection prevents competitors from launching a similar drug for the same use during that period. Additional benefits include tax credits for qualified clinical trial costs, a waiver of the substantial Prescription Drug User Fee Act (PDUFA) fees required for a new drug application, and eligibility for FDA grants and clinical protocol assistance.

These incentives are critical for encouraging pharmaceutical companies to invest in conditions that may not have the blockbuster market size of more common diseases, ensuring that patients with rare but serious conditions are not left behind.

The Path Forward: From Licensing to Clinical Trials

Pegrizeprument's journey to this point is a testament to modern pharmaceutical collaboration. The drug, also known by its original name FR104, was initially developed by OSE Immunotherapeutics, a French biotechnology firm. In April 2021, Veloxis, a subsidiary of the Japanese conglomerate Asahi Kasei, licensed the worldwide rights to develop, manufacture, and commercialize the agent for all transplant indications. The deal involved an upfront payment of €7 million to OSE, with the potential for an additional €315 million in milestone payments, plus royalties on future sales.

This partnership leverages OSE's expertise in immunology with Veloxis's established focus and commercial infrastructure in the transplant market. Veloxis has already begun advancing the drug's clinical program. While the new Orphan Drug Designation is for liver transplantation, the most advanced ongoing studies are in kidney transplant recipients, a common strategy for drugs with potential across multiple organ types.

A Phase 1/2 study (FIRsT) in kidney transplant patients has been completed, reportedly showing a good safety profile and providing the data needed to move forward. Veloxis is now sponsoring a larger Phase 2 trial, RENGEVITY-201, designed to evaluate the safety and efficacy of pegrizeprument compared to the standard-of-care drug tacrolimus in kidney transplant recipients. While not yet recruiting, the study is a critical next step in proving the drug's value.

The Orphan Drug Designation for liver transplant patients strongly suggests that Veloxis will be expanding its clinical program to include this population, building on the data generated from its kidney transplant studies. Success in the clinic could position pegrizeprument as a foundational therapy in a new era of transplant medicine, one focused on targeted immunomodulation that promises not only to save lives but also to vastly improve them.