FDA Approves Soliris for Rare Pediatric Blood Disorder

The FDA has approved Soliris to treat patients with atypical Hemolytic Uremic Syndrome that can lead to kidney failure and is also associated with increased risk of death and stroke.

The U.S. FDA has approved Soliris (eculizumab) to treat patients with atypical Hemolytic Uremic Syndrome (aHUS), a rare and chronic blood disease that can lead to kidney (renal) failure and is also associated with increased risk of death and stroke.

The FDA first approved Soliris in March 2007 to treat paroxysmal nocturnal hemoglobinuria (PNH), a rare type of blood disorder that can lead to disability and premature death. 

There are no other FDA-approved treatments for aHUS, and the safety and effectiveness of current standard treatment, plasma therapy (plasma exchange or fresh frozen plasma infusion), have not been studied in well controlled trials.

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