Only about one in a million persons are diagnosed with PNH. However, this rare disorder that usually develops in adults is characterized by abnormally developed red blood cells that can lead to disability and premature death.
Soliris can help treat the symptoms of PNH, but it is not a cure.
The drug is classified by the FDA as an "orphan drug," not previously marketed in the U.S.
Soliris, manufactured by Alexion Pharmaceuticals Inc. (Cheshire, CT), treats the breakdown of red blood cells (the disease's most common characteristic), and the drug can destroy the PNH red blood cells, but can not cure the disease.
This disease causes anemia when left untreated, which is usually evident by abnormally darkened urine. Several life-threatening consequences can result from PNH, including fatal strokes, heart attacks and intestinal disease, according to the FDA.
Patients with severe PNH frequently experience pain, become easily fatigued, can become incapacitated with weakness, form blood clots, and usually require frequent blood transfusions, per the FDA.
"This product is important in that it offers a treatment other than blood transfusion[s] that may help this small population of patients who are often very ill," stated Steven Galson, M.D., M.P.H., who is the director for the FDA's Center for Drug Evaluation and Research.
"This approval is one of multiple examples of how the orphan products program can benefit the public health with urgently needed products that would otherwise not be commercially available," Dr. Galson adds.
The FDA notes that the drug Soliris blocks the body's natural immune system, which can affect the patient's susceptibility to some serious infections, in particular bacterial meningitis. Therefore, due to the high risk of meningitis infections, patients are warned to obtain vaccinations before using Soliris.
The FDA has required the manufacturer to label the drug and medication guide literature to alert patients and physicians to this warning. In addition a physician educational program has been instituted for this orphan drug.
Soliris is considered an orphan drug because it treats a rare disease/condition that affects fewer than 200,000 people in the U.S., according to the FDA's press release.
"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," reports the FDA.
