Veloxis’ VEL-101 Granted FDA Orphan Drug Designation for Liver Transplant Rejection Prevention

Veloxis Pharmaceuticals, Inc., a global specialty pharmaceutical company and part of Asahi Kasei Corporation, has received Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for pegrizeprument (VEL-101).

The designation covers the prevention of organ rejection in patients undergoing liver transplantation, marking an important regulatory milestone in the drug’s development.

Addressing a persistent unmet need in liver transplantation

Orthotopic liver transplantation (OLT) is a life-saving intervention for patients with:

• Acute liver failure
• Chronic liver disease
• Primary hepatic malignancies

However, transplant recipients require lifelong immunosuppression, which often leads to:

• Complex dosing regimens
• Long-term toxicities
• Increased infection risk
• Allograft rejection

Despite advances in post-transplant care, immunosuppression-related complications remain a major cause of graft failure and reduced patient survival, highlighting the need for safer, more targeted therapies.

Why Orphan Drug Designation matters?

The FDA’s Orphan Drug Designation is granted to investigational therapies intended to treat rare diseases affecting fewer than 200,000 patients in the US. The designation provides several development incentives, including:

• Regulatory support during development
• Eligibility for market exclusivity upon approval
• Reduced barriers for advancing novel therapies in underserved indications

For Veloxis, the designation reinforces the clinical and regulatory relevance of pegrizeprument in liver transplantation.

Management commentary

“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.
“This Orphan Drug Designation represents an important step forward for pegrizeprument and reinforces our commitment to advancing innovative therapies for transplant patients.”

About pegrizeprument (VEL-101)

Pegrizeprument is a pegylated monoclonal antibody fragment designed to deliver selective immunosuppression.

Mechanism of action

VEL-101 works through a dual immunomodulatory mechanism:

• Directly blocks CD28-mediated T-cell costimulation, preventing effector T-cell activation
• Preserves CTLA-4 signaling, a natural immune checkpoint that helps regulate immune responses

This differentiated approach aims to maintain effective immunosuppression without fully suppressing immune regulation, potentially reducing toxicity and long-term complications.

Development background

• Pegrizeprument is also known as FR104
• Veloxis licensed the asset from OSE Immunotherapeutics in April 2021
• The agreement granted Veloxis worldwide rights to develop, manufacture, and commercialize the drug across all transplant indications

VEL-101 is currently being developed for the prevention of acute rejection in solid organ transplant recipients.

About Veloxis Pharmaceuticals

Veloxis Pharmaceuticals, an Asahi Kasei company, is focused on improving outcomes for transplant patients worldwide.

• Headquarters: Cary, North Carolina, USA
• Core focus: Development and commercialization of medicines for transplant and related serious conditions

About Asahi Kasei

Founded in 1922, Asahi Kasei Group operates across Healthcare, Homes, and Materials, employing over 50,000 people globally.

Its healthcare business spans:

• Pharmaceuticals
• Biotherapeutics
• Critical care devices and systems

The group’s mission centers on delivering solutions that support sustainable health and living worldwide.

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