Pharming Group NV has received orphan drug designations for recombinant human C1 inhibitor (rhC1INH) from the Food and Drug Administration (FDA).
The company has obtained designations on rhC1INH for two separate disease indications - the prevention and/or the treatment of Delayed Graft Function (DGF) after solid organ transplantation and the treatment of Capillary Leakage Syndrome (CLS), stated a company release.
Over 25,000 solid organs were transplanted in the US in 2005, including kidney, liver, lung and heart transplants. 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.
Over 100,000 patients in the US develop Capillary Leakage Syndrome annually as 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.
"Pharming's rhC1INH product could provide new treatments for immune mediated diseases such as Delayed Graft Function in organ transplantation and Capillary Leakage Syndrome, conditions with a high burden and limited treatment options for patients," said Dr Francis Pinto, CEO of Pharming. "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."
The FDA Orphan Drug designation is reserved for promising new therapies being developed to treat diseases that affect fewer than 200,000 people in the United States. This designation provides an accelerated review process, tax advantages and a seven-year period of market exclusivity in the US upon product approval. Pharming also has an Orphan Drug designation on rhC1INH for the treatment of Hereditary Angioedema.