FDA approves Soliris for Hemolytic Uremic Syndrome

The FDA has approved Soliris (eculizumab) from Alexion Pharma for the treatment of patients with atypical Hemolytic Uremic Syndrome (aHUS), a rare, chronic blood disease that can lead to kidney failure, stroke, heart attack and death. This is the first treatment for aHUS that has been FDA-approved and the first approval for use of Soliris in children. The decision is based on two single-arm trials consisting of 37 adults and adolescents with aHUS and one retrospective investigation involving 19 pediatric patients and 11 adult patients with aHUS. This approval comes with an extension of the existing Risk Evaluation and Mitigation Strategy (REMS), to ensure health care professionals and patients are aware of the associated risk of life-threatening meningococcal infections.