FDA approves first-ever drug for rare blood disorder

SILVER SPRING, Md. — The Food and Drug Administration has approved a treatment made by Alexion Pharmaceuticals for a rare blood disorder that affects children.

The FDA announced the approval of Soliris (eculizumab) for atypical hemolytic uremic syndrome, a rare and chronic disease that can lead to kidney failure, stroke and death. According to the FDA, aHUS accounts for 5% to 10% of all cases of hemolytic uremic syndrome.

"This is the first approval of a drug for treating this life-threatening disease, and the first approval for use of Soliris in children," FDA Office of Hematology and Oncology Products Richard Pazdur said. "This approval underscores how an increased understanding of the biology of the disease and how a drug interacts with that process can expedite drug development."


Interested in this topic? Sign up for our weekly Retail Health Provider e-newsletter.