FDA okays orphan drug Soliris for deadly blood disease

red blood cells
AP
Red blood cells
AP

(CBS) The FDA has approved the drug Soliris (eculizumab) for the treatment of a rare and potentially deadly blood disease known as atypical hemolytic uremic syndrome (aHUS).

There are no other FDA-approved treatments for aHUS, in which the formation of clots within blood vessels can "shatter" blood cells, leading to kidney failure or stroke. Children are at greatest risk for the disease, according to website of the Foundation for Children with Atypical HUS.

"This is the first approval of a drug for treating this life-threatening disease, and the first approval for use of Soliris in children," Dr. Richard Pazdur, director of the agency's Office of Hematology and Oncology Products, said in a written statement issued by the FDA.

Soliris, which is marketed by Alexion Pharmaceuticals of Cheshire, Conn., works by blocking the action of proteins that are believed to play a role in aHUS. It's classified as an orphan drug, a term used to indicate drugs that have shown promise for the diagnosis or treatment of rare diseases. Doctors who wish to prescribe the drug must enroll in a registration program, according to the statement.

AHUS is extremely rare. There are about 300 cases in the U.S., along with an unknown number of cases around the world, according to foundation's website.