Pharming gets orphan-drug status for rhC1INH for two transplant-related disorders

By E. Janene Geiss

Philadelphia, June 14 - Pharming Group NV said Wednesday it has received orphan-drug designations for recombinant human C1 inhibitor (rhC1INH) from the Food and Drug Administration.

The company has obtained designations on rhC1INH for two separate disease indications - the prevention and/or the treatment of delayed graft function after solid organ transplantation and the treatment of capillary leakage syndrome (CLS), according to a company news release.

More than 25,000 solid organs were transplanted in the United States in 2005, including kidney, liver, lung and heart transplants.

Delayed graft function (DGF) 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, officials said.

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, officials said.

C1 inhibitor has been shown in numerous models of organ transplantation to improve early graft function, the company said.

More than 100,000 patients in the United States 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, officials said.

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 United States upon product approval.

Pharming also has an orphan-drug designation on rhC1INH for the treatment of hereditary angioedema.

Pharming is a biopharmaceutical company based in Leiden, The Netherlands.

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