Pharming transplant therapy receives orphan designation

The compound won the designation for the prevention and/or treatment of delayed graft function (DGF) after solid organ transplantation and the treatment of capillary leakage syndrome (CLS).

Delayed graft function is a common complication affecting all solid organs in the post-transplant period. DGF results in significant morbidity and mortality from early graft dysfunction and from decreased long- term graft survival. The condition also prolongs hospitalization and requires substitute therapies for these patients, such as dialysis or ventilatory support.

DGF remains a critical unmet medical need despite improvements in immunosuppression, organ preservation, and surgical technique. C1 inhibitor has been shown in numerous models of organ transplantation to improve early graft function.

Capillary leakage syndrome is a complication of various disease states, including bone marrow/stem cell transplantation, IL-2 therapy, sepsis, and neonatal cardiac surgery. CLS is a severe life-threatening condition characterized by excessive fluid loss into the tissue space, which can result in hemodynamic instability, pulmonary edema, ascites, and death.

Current therapies for patients with CLS are limited to supportive care and treatment of the underlying condition. Previous clinical work has demonstrated that C1 inhibitor may be an effective anti-inflammatory that can control the mechanisms contributing to CLS.

“The Orphan Drug designations from the FDA further validate the potential of rhC1INH as an innovative therapy and are a significant achievement as we advance development of rhC1INH for these indications,” said Dr Francis Pinto, CEO of Pharming.