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In one of the first rays of hope for sufferers of hemolytic uremic syndrome (HUS), FDA announced today its approval of Soliris “… 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.”

According to FDA, atypical HUS accounts for 5-10% of all cases of hemolytic uremic syndrome and affects children disproportionately. Soliris is the first drug to be approved in the USA for treating atypical HUS.

Soliris (eculizumab) is a humanized monoclonal antibody that was first approved by FDA in 2007 to treat a rare type of blood disorder (paroxysmal nocturnal hemoglobinuria), and is classified as an orphan drug. It is marketed in the USA by Alexion Pharmaceuticals.

While today’s FDA approval of Soliris is limited to atypical HUS, the agency’s action represents the first hint that a specific treatment for more common forms of hemolytic uremic syndrome may be in the offing. HUS is a life-threatening syndrome that affects a significant percentage of people – especially children – who are infected with E. coli O157:H7 and other shiga-toxin producing strains of E. coli.

More than 900 victims of this summer’s E. coli O104:H4 outbreak developed HUS, and 34 of those people died of the disease. The death toll might have been even higher, had doctors not administered eculizumab (Soliris) to 3 children with severe HUS and central nervous system complications. The children were at risk of permanent neurologic damage and not responding to conventional treatment.

All three children responded to the Soliris treatment, and eventually were discharged from hospital with apparently normal neurological status and little or no residual effects of their illness. Six months later, all three children were “in full remission,” according to a report published in New England Journal of Medicine.

Doctors who want to use Soliris to treat HUS must jump through a few extra hoops, due to the limited nature of its clinical trial. Soliris is available only through a restricted program, according to FDA, and prescribers must enroll in a registration program and provide a medication guide to patients who receive the drug.

Nevertheless, today’s approval is major news for HUS sufferers, so many of whom are children. Parents whose children are suffering from HUS – and physicians who treat HUS patients – should educate themselves about this new treatment possibility.

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