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FDA Grants Orphan Drug Designation to Soliris®

Tue, 01/21/2014 - 9:00am

Alexion Pharmaceuticals has announced that the U.S. Food and Drug Administration (FDA) has granted an orphan drug designation (ODD) to Soliris ® (eculizumab), a first-in-class terminal complement inhibitor, for the prevention of delayed graft function (DGF) in renal transplant patients. DGF is an early and serious complication of organ transplantation that is characterized by the failure of a transplanted organ to function normally immediately following transplantation. In patients undergoing a kidney transplant, DGF leads to the patient requiring dialysis in order to survive. 1-3

Soliris is currently approved for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), two debilitating, ultra-rare and life-threatening disorders caused by chronic uncontrolled complement activation. Soliris is not approved in any country to prevent or treat DGF following kidney or other solid organ transplantation.

“For kidney transplant patients with increased risk, there is a serious unmet medical need for a treatment to prevent delayed graft function and its harmful consequences,” said Martin Mackay, Ph.D., Executive Vice President, Global Head of R&D at Alexion. “By specifically inhibiting the terminal complement pathway, which is believed to play a critical role in the development of DGF, Soliris has the potential to lower the risk of DGF, a benefit that may have positive implications for longer-term kidney function and clinical outcomes for patients. In addition, a significant number of donor kidneys are reportedly never used and thus discarded each year due to the risk of poor outcomes associated with DGF, therefore reducing the risk of DGF may enable more patients to receive a kidney transplant.”

The FDA, through its Office of Orphan Products Development (OOPD), grants orphan status to drugs and biologic products that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S. Orphan drug designation provides a drug developer with certain benefits and incentives, including a period of marketing exclusivity if regulatory approval is ultimately received for the designated indication.

Alexion plans to initiate a single multinational DGF registration study with Soliris later this year. Alexion looks forward to working closely with the FDA to gather the clinical evidence needed to support approval for this indication.

About Delayed Graft Function (DGF)

DGF is an early and serious complication of organ transplantation that is characterized by the failure of a transplanted organ to function normally immediately following transplantation. In the case of DGF in the setting of kidney transplantation, the patient requires dialysis after the transplantation procedure. 1-3 Most often, DGF results from organ injury caused by reduction and/or restoration of blood flow, and the associated inflammation, including complement activation. 1-4 DGF has a substantial negative impact on graft function both in the short and long term, which can result in premature graft loss, prolonged hospitalization or patient death. 5,6 In addition, as kidney donors are in short supply, reducing the risk of DGF may allow more donor kidneys to be transplanted. At present, 15-20 percent of donor kidneys are reportedly never used and thus discarded each year in the U.S. and Europe due to the risk of poor outcomes associated with DGF, 7,8 denying many patients the benefit of solid organ transplantation.

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