FRIDAY, Sept. 23 (HealthDay News) -- Soliris (eculizumab) has
been approved by the U.S. Food and Drug Administration as the first
drug to treat atypical hemolytic uremic syndrome (aHUS), a rare
blood disease that may trigger kidney failure, stroke or death.
Most people with aHUS are children, the FDA said in a news
release. The atypical form affects up to 10 percent of all cases of
hemolytic uremic syndrome.
Solaris was first approved in 2007 to treat another rare blood
disorder, paroxysmal nocturnal hemoglobinuria (PNH). In people with
aHUS, the drug limits the activities of proteins that play a role
in the disease, the FDA said.
Soliris has been classified as an orphan drug, the agency said,
meaning the drug has demonstrated promise in treating rare diseases
In clinical testing of people with aHUS, Soliris' most common
side effects included high blood pressure, diarrhea, headache,
anemia, nausea and vomiting, infections of the upper respiratory
tract and urinary tract, and a decrease in white blood cells.
The drug may also increase users' risk of life-threatening
meningococcal infections. It will continue to be made available
only via a restricted delivery program, under which prescribers
must register with the FDA, the agency said.
Soliris is marketed by Alexion Pharmaceuticals, based in
The FDA has
more about this approval.