FDA Approves Alexion’s Soliris To Treat Rare Blood Disease

The Food and Drug Administration Sept. 23 approved Soliris (eculizumab) to treat pediatric and adult patients with atypical hemolytic uremic syndrome (aHUS), a rare and chronic blood disease that progressively damages vital organs, leading to stroke, heart attack, kidney failure, and death.

Soliris is a targeted therapy that works by inhibiting proteins that play a role in aHUS, FDA said. The drug is marketed by Alexion Pharmaceuticals.

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