WASHINGTON (AP) – The first drug to treat a blood disorder that strikes one in a million people won federal approval Friday.
Soliris, or eculizumab, treats paroxysmal nocturnal hemoglobinuria or PNH, the Food and Drug Administration said. The rare blood disorder can lead to disability and premature death.
The drug is made by Alexion Pharmaceuticals Inc. of Cheshire, Conn.
The drug is not a cure. Instead, it treats the breakdown of abnormally developed red blood cells that are the hallmark of the disease.
If left untreated, the body naturally breaks down those cells, causing anemia. Depending on the severity of the disease, patients with PNH may have pain, fatigue and debilitating weakness and require frequent blood transfusions.
It also can lead to blood clots and life-threatening or fatal strokes, heart attacks and intestinal disease, the FDA said.
Since Soliris blocks the body’s natural immune system it can increase susceptibility to serious infections, particularly bacterial meningitis, the FDA said.
The drug’s label flags that risk and requires that patients be vaccinated for meningitis before starting treatment.
Alexion, which has about 300 employees worldwide, has also applied to sell the drug in South America, Japan, Australia and 25 European countries.
Dr. Leonard Bell, Alexion’s chief executive officer and a former Yale University cardiologist and pathologist, said this week that the drug has been in development for about 15 years.
“In many ways, it’s the end of a long path,” he said. “We’re very excited to now be able to move the drug and make it available to patients. It’s really the commencement of a new phase.”
Bell said the company has not determined the cost to consumers. He expects about 500 to 1,500 people will be treated with it each year, primarily by private hematologists and oncologists.
“We expect a lot out of Soliris, and we expect revenues to be able to fund other innovations,” he said. “It’s extremely exciting. I think it’s an excellent example of a small business of scientists focusing on a rare disease.”
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