The FDA has approved Soliris to treat patients with atypical Hemolytic Uremic Syndrome, a rare and chronic blood disease that can lead to kidney (renal) failure and is also associated with increased risk of death and stroke.
Atypical HUS accounts for 5 to 10% of all cases of hemolytic uremic syndrome. The disease disproportionately affects children.
Soliris is a targeted therapy that works by inhibiting proteins that play a role in aHUS. The FDA first approved Soliris in March 2007 to treat paroxysmal nocturnal hemoglobinuria (PNH), a rare type of blood disorder that can lead to disability and premature death. Soliris is classified as an orphan drug. Orphan drugs are those that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions.
There are no other FDA-approved treatments for aHUS, and the safety and effectiveness of current standard treatment, plasma therapy (plasma exchange or fresh frozen plasma infusion), have not been studied in well controlled trials.
Soliris' safety and effectiveness were established in two 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 that 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.
The most common side effects seen in patients treated with Soliris for aHUS included high blood pressure (hypertension), diarrhea, headache, anemia, vomiting, nausea, upper respiratory and urinary tract infections, and a decrease in white blood cells (leukopenia).