Paroxysmal nocturnal hemoglobinuria (PNH), which usually develops in adults, is a disease characterized by red blood cells that develop abnormally. Once the abnormal cells are present in the bloodstream, naturally occurring proteins (called the complement system), designed to destroy bacteria and other infection-causing organisms, break these cells down. This leads to abnormally darkened urine and, more importantly, causes anemia. Depending upon the severity of the disorder, patients with PNH may have pain, fatigue and debilitating weakness, the need for frequent blood transfusions, blood clots, and life-threatening or fatal strokes, heart attacks and intestinal disease. Soliris does not cure PNH, but treats the breakdown of red blood cells, the most common characteristic of PNH. Soliris acts to block the complement system activity, including the destruction of PNH red blood cells. Terminal complement inhibition with Soliris increases the susceptibility to serious meningococcal infections.
The U.S. Food and Drug Administration (FDA) approved Soliris on March 16, 2007. Soliris is classified as an orphan drug product. Orphan products are developed to treat rare diseases or conditions that affect fewer than 200,000 people in the United States. The Orphan Drug Act provides a seven-year period of exclusive marketing to the first manufacturer who obtains marketing approval for a designated orphan product. About one person out of a million people will be diagnosed with PNH.
Atypical Hemolytic Uremic Syndrome (aHUS)
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 percent of all cases of hemolytic uremic syndrome. The disease disproportionately affects children. 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. (7)
Soliris is given through intravenous (IV) infusion. The recommended dose of Soliris is dependent on the indication for which the drug is administered and the age of the patient (adult or child). Soliris is manufactured by Alexion Pharmaceuticals, Inc.