Veloxis Pharmaceuticals announced that pegrizeprument, a novel investigational maintenance immunosuppressive agent being developed for prevention of acute rejection in solid organ transplant recipients, has been granted Orphan Drug Designation (ODD) by the FDA for prevention of organ rejection in patients receiving a liver transplant. This new designation is a critical step for Veloxis in the development of pegrizeprument and reinforces the unmet need for orthotopic liver transplantation (OLT) patients around the world.
Liver transplantation can be a life-saving treatment for patients with acute liver failure, chronic liver failure, or primary hepatic malignancy. However, OLT patients require life-long immunosuppression, often associated with complex dosing regimens and comorbid toxicities. Despite advancements in post-transplant clinical care, complications of immunosuppression and allograft rejection may negatively impact graft and patient survival. Therefore, a significant unmet need remains for new therapies that address these complications while maintaining adequate immunosuppression.
"We are pleased by the FDA's decision, which highlights the need for groundbreaking therapies to improve outcomes for liver transplant recipients," says Stacy Wheeler, CEO of Veloxis. "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."
In the absence of new, novel immunosuppressant options, the Orphan Drug Designation reflects the potential for pegrizeprument to address a critical need for OLT recipients. Veloxis continues to research how pegrizeprument may be able to address unmet needs in the solid organ transplantation population.
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