OSE Immunotherapeutics: Pegrizeprument Granted Orphan Drug Status in the United States
The FDA has granted orphan drug designation to pegrizeprument (VEL-101) for the prevention of organ rejection in patients undergoing liver transplantation, as announced by Veloxis Pharmaceuticals, a partner of OSE Immunotherapeutics.
FDA Designation Announced
Veloxis Pharmaceuticals, a partner of the company, announced that the U.S. Food and Drug Administration has granted pegrizeprument, also known as VEL-101, orphan drug designation for the prevention of organ rejection in liver transplantation, according to a statement from OSE Immunotherapeutics. This status, granted by the FDA, applies to experimental drugs and biological products intended to prevent, diagnose, or treat rare diseases affecting fewer than 200,000 people in the United States. According to the company, this designation aims to encourage the development of treatments for patients with traditionally underserved diseases. Pegrizeprument is an immunomodulatory monoclonal antibody fragment initially discovered and developed by OSE Immunotherapeutics, then licensed to Veloxis in 2021 for all transplantation-related indications.
Mechanism of Action
Pegrizeprument is a pegylated monoclonal antibody fragment that binds to CD28 and blocks the co-stimulation of effector T cells mediated by CD28, without blocking CTLA-4, a protein on the surface of T cells that helps regulate the body's immune responses, the group indicates. VEL-101 is therefore expected to have a dual mechanism of action, according to the company: it directly blocks CD28-mediated T cell activation and indirectly allows for CTLA-4 mediated immunosuppressive functions. The product is currently in development to prevent rejection after kidney transplantation, according to OSE Immunotherapeutics. Pegrizeprument, also known under the names VEL-101 and FR104, was licensed by Veloxis Pharmaceuticals from OSE Immunotherapeutics in April 2021.
Licensing Agreement Details
As part of the licensing agreement concluded in April 2021, Veloxis Pharmaceuticals obtained global rights to develop, manufacture, and market pegrizeprument for all transplantation indications, according to the statement. Veloxis is responsible for the global development, manufacturing, and future commercialization of the product, OSE Immunotherapeutics indicates. Sonya Montgomery, Chief Development Officer of OSE Immunotherapeutics, stated that this designation highlights the need for improved options in solid organ transplantation and represents a significant milestone in the development of pegrizeprument. OSE Immunotherapeutics is a biotechnology company based in Nantes and Paris, listed on Euronext, which develops products in immuno-oncology and immuno-inflammation.
