News Feature | January 22, 2014

The FDA Granted Soliris With An Orphan Drug Designation

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By Cassandra Leger

Alexion Pharmaceuticals announced that the FDA granted the drug Soliris® (eculizumab) with an orphan drug designation. This designation is given to drugs that are developed to treat rare conditions that affect fewer than 200,000 individuals. Soliris is used to prevent delayed graft function (DGF) in patients who undergo kidney transplant surgery. DGF‘s onset is immediate and refers to the new organ not responding or functioning properly immediately after it has been introduced to the body.

Currently Soliris has been approved to treat other indications such as paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). Both are debilitating conditions that arise from uncontrolled complement activation and can pose a life-threatening risk to patients.

Dr. Martin Mackay, Ph.D., Executive Vice President, Global Head of R&D at Alexion, stated "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.” He went on to say, "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."

Alexion has made plans to conduct a multinational study using Soliris in late 2014.