Soliris (eculizumab; Alexion), an orphan drug, has been approved for the treatment of atypical hemolytic uremic syndrome (aHUS). This approval was based on data from two Phase 2 single-arm trials in 37 adults and adolescent patients with aHUS and one retrospective study in 19 pediatric patients and 11 adult patients with aHUS. Patients treated with Soliris in these studies experienced a favorable improvement in kidney function, including elimination of the requirement for dialysis in several patients with aHUS who did not respond to plasma therapy. Patients treated with Soliris also exhibited improvement in platelet counts and other blood parameters that correlate with aHUS disease activity.
Soliris is already approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Soliris will continue to be available only through a restricted program, and prescribers must enroll in a registration program and provide a medication guide to patients who receive the drug.
For more information call (888) SOLIRIS or visit www.soliris.net.