FDA Grants Orphan Drug Status to Veloxis' Pegrizeprument for Liver Transplant Rejection Prevention

Event summary

  • FDA granted Orphan Drug Designation to pegrizeprument (VEL-101) for preventing organ rejection in liver transplant patients on January 21, 2026.
  • Pegrizeprument is a novel immunomodulatory monoclonal antibody fragment originally developed by OSE Immunotherapeutics and licensed to Veloxis in 2021.
  • The drug is currently in development for kidney transplantation prevention (NCT07290777).
  • Veloxis holds worldwide rights to develop, manufacture, and commercialize pegrizeprument for all transplant indications.

The big picture

The FDA's Orphan Drug Designation for pegrizeprument highlights the growing focus on developing specialized treatments for rare conditions in transplant medicine. This designation could provide Veloxis with significant commercial advantages, including tax credits, waived FDA fees, and seven years of market exclusivity if approved. The strategic partnership between OSE Immunotherapeutics and Veloxis underscores the importance of collaborative innovation in addressing unmet medical needs in organ transplantation.

What we're watching

Clinical Progress
The pace at which pegrizeprument advances through clinical trials will determine its market potential in transplant medicine.
Regulatory Strategy
Whether Veloxis can leverage the Orphan Drug Designation to accelerate FDA approval and secure market exclusivity.
Commercialization Path
How Veloxis plans to position pegrizeprument against existing and emerging therapies in the transplant rejection prevention space.