The U.S. Food and Drug Administration approved the drug Soliris (eculizumab) by Alexion Pharmaceuticals Inc. to treat a rare genetic blood disease in adults and children.
The medicine treats atypical hemolytic uremic syndrome (aHUS), an ultra-rare, life-threatening, genetic disease that progressively damages vital organs, leading to stroke, heart attack, kidney failure and death.
"The FDA approval of Soliris in aHUS marks the most important advance that has been made for patients and families with this disease," said said Larry Greenbaum, M.D., Ph.D., Director of Pediatric Nephrology at Emory University and Children's Healthcare of Atlanta in a statement published by Alexion.
Despite current supportive care, more than half of all patients with aHUS die, require kidney dialysis or have permanent kidney damage within 1 year of diagnosis, Alexion said.
Soliris directly targets uncontrolled complement activation, the underlying cause of the progressive organ failure and shortened life span of patients with aHUS.
The drug markedly decreased the thrombotic microangiopathy (TMA) process, which is responsible for thrombosis, renal impairment, seizures, and angina in patients with aHUS, according to Dr. Craig Langman of Northwestern University.
Published by Medicaldaily.com
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