Reporting from Washington — The Food and Drug Administration on Wednesday approved Benlysta as the first new drug treatment for lupus in 56 years, offering possible relief from painful symptoms of the sometimes fatal autoimmune disease.
Government approval also sets the stage for possible blockbuster sales for the drug's developer, Human Genome Sciences Inc.
The decision was widely expected after an FDA advisory panel voted 13 to 2 in November to recommend approval of the drug.
"It's a historic day. It really does open a door," said Sandra Raymond, president of the Lupus Foundation of America. "Not that Benlysta is a miracle drug. It's not. I think the lupus community is sanguine about that. But they know that there are other companies coming behind" Human Genome.
The drug is likely to cost at least $30,000 a year in the U.S. and $23,000 overseas, said Avik Roy, who follows the company for Monness, Crespi, Hardt & Co. It has potential global sales of $5 billion, with almost $3 billion of that coming from the U.S., he said.
With an autoimmune disease such as lupus, the body's immune system can't tell the difference between healthy substances and harmful ones and therefore attacks tissues and organs. It usually appears in people between ages 15 and 44, with women nine times as likely as men to develop the disease.
Symptoms vary widely, making the disease hard to diagnose and treat. Symptoms can include joint pain, fatigue, fever, stiffness, mouth sores, hair loss and rashes. The disease can attack the kidney, lungs and other internal organs, leading to death.
Estimates on the number of lupus patients in the U.S. vary widely, ranging from 300,000 to 1.5 million, the FDA said.
One of Benlysta's benefits is that patients can use less prednisone, a steroid used to control symptoms, said Cynthia Aranow, co-director of autoimmune diseases and clinical research at the Feinstein Institute for Medical Research in Manhasset, N.Y.
"The ability to be steroid-sparing is very important" because prednisone has numerous troubling side effects including osteoporosis, diabetes and high blood pressure, Aranow said.
Benlysta, known generically as belimumab, will be administered intravenously in a doctor's office or other outpatient setting.
When used in combination with other treatments, Benlysta "may be an important new treatment approach for healthcare professionals and patients looking to help manage symptoms associated with this disease," Curtis Rosebraugh, a senior official in the FDA's drug evaluation office, said in a statement.
African Americans suffer from the disease at higher rates than other races.
Two clinical studies that demonstrated the safety and effectiveness of Benlysta showed that African Americans and Africans did not appear to respond to the drug. The FDA said the small number of participants from those two groups could have affected the results, and Human Genome has agreed to further study.
Benlysta is the first FDA-approved drug for Human Genome, an 18-year-old biotech firm in Rockville, Md.
"We expect to have this novel therapy available to physicians and patients within about two weeks, and our entire organization looks forward to the positive impact we hope this new therapy will have for patients with systemic lupus," Human Genome President and Chief Executive H. Thomas Watkins said in a statement.
The company is scheduled to split the profits with its marketing partner, GlaxoSmithKline.
The approval assures Human Genome years of healthy revenue because Benlysta has no competitors and is not likely to encounter many because it's a biologic drug, a category of pharmaceuticals that is exceedingly hard to manufacture, Roy said.
"They're part of a very small club of biotech companies with an original biologic that will be on the market for a long time," Roy said.
Roy said shares of Human Genome had traded as low as 45 cents, but the stock closed at $25.68 on Wednesday before the FDA announcement.
Besides Benlysta and prednisone, only two other drugs are approved for treating lupus — aspirin and Plaquenil, an antimalarial drug. Both were approved during the 1950s and can have side effects, including stomach bleeding for aspirin and headache, dizziness and nausea for Plaquenil.
Times staff writer Thomas H. Maugh II contributed to this report.