Veloxis Pharmaceuticals (Veloxis), a partner of OSE Immunotherapeutics, has announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to pegrizeprument (VEL-101), the company's novel investigational maintenance immunosuppressive agent for the prevention of heart allograft rejection.
This follows the recent ODD granted in January to prevent organ rejection in liver transplant patients.
Pegrizeprument, also known as VEL-101 and FR104, is a novel investigational immunomodulatory monoclonal antibody fragment.
It was originally discovered and developed by OSE Immunotherapeutics for various transplant-related indications.
In April 2021, Veloxis licensed pegrizeprument from OSE Immunotherapeutics.
As part of this licensing agreement, Veloxis gained worldwide rights to develop, manufacture and commercialise pegrizeprument for all transplant indications.
"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," said 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.
Why this matters
Orthotopic heart transplantation is a life-saving procedure for heart failure patients, but recipients require lifelong immunosuppression, which can lead to complications and toxicities.
These issues can negatively impact graft and patient survival. Thus, there is a significant need for new therapies that address these challenges while maintaining adequate immunosuppression.
Veloxis is committed to developing pegrizeprument as a novel, targeted therapy to fill these critical gaps in long-term post-transplant care.