For the first time since 1955, the United States Food and Drug Administration (FDA) has approved a new drug for the treatment of lupus. In a release dated March 9, 2011, the federal agency noted that Benlysta ® is approved for patients with active, autoantibody-positive lupus who are receiving standard therapy, including corticosteroids, antimalarials, immunosuppressives, and nonsteroidal anti-inflammatory drugs.
The FDA noted that the drug, “is delivered directly into a vein (intravenous infusion) and is the first inhibitor designed to target B-lymphocyte stimulator (BLyS) protein, which may reduce the number of abnormal B cells thought to be a problem in lupus.”
The online information service of the Mayo Clinic defines lupus as “a chronic inflammatory disease that occurs when a body’s immune system attacks its own tissues and organs. Inflammation caused by lupus can affect many different body systems, including joints, skin, kidneys, blood cells, heart and lungs.” It further notes that “lupus occurs more frequently in women, though it isn’t clear why.”
A 2008 study referenced in media reports announcing the approval of the drug noted that the most common form of lupus affects between 161,000 and 322,000 Americans. People of all races can have the disease; however, African American women have a 3 times higher incidence than Caucasian women. The disease usually strikes younger women of child-bearing age.
A Big Win for Human Genome Sciences
The approval of Benlysta represents a major victory for Human Genome Sciences Inc., a small biotechnical company that has been in existence for only 18 years. This will be the first major product that the company has brought to market and with the help of its considerably larger distribution partner, GlaxoSmithKline PLC, Wall Street pharmaceutical analysts and medical practitioners predict the drug could be a “blockbuster.”
“Benlysta, when used with existing therapies, may be an important new treatment approach for health care professionals and patients looking to help manage symptoms associated with this disease,” said Curtis Rosebraugh, M.D., M.P.H., director of the Office of Drug Evaluation II in the FDA’s Center for Drug Evaluation and Research.
In announcing the approval of the drug, the FDA noted that “Two clinical studies involving 1,684 patients with lupus demonstrated the safety and effectiveness of Benlysta. The studies diagnosed patients with active lupus and randomized them to receive Benlysta plus standard therapy, or an inactive infused solution (placebo) plus standard therapy. The studies excluded patients who had received prior B-cell targeted therapy or intravenous cyclophosphamide, and those who had active lupus involving the kidneys or central nervous system.”
The federal agency continued, “Patients treated with Benlysta and standard therapies experienced less disease activity than those who received a placebo and standard of care medicines. Results suggested, but did not definitively establish, that some patients had a reduced likelihood of severe flares, and some reduced their steroid doses.
“African American patients and patients of African heritage participating in the two studies did not appear to respond to treatment with Benlysta. The studies lacked sufficient numbers to establish a definite conclusion. To address this concern, the sponsor has agreed to conduct an additional study of people with those backgrounds to further evaluate the safety and effectiveness of Benlysta for this subgroup of lupus patients.”
Additional caveats of the FDA’s approval noted that those receiving Benlysta during clinical studies “reported more deaths and serious infections compared with placebo. The most common side effects in the studies included nausea, diarrhea, and fever. It found that patients also commonly experienced infusion reactions, and recommended that pre-treatment with an antihistamine should be considered.”
What This Means for the Drug Companies
This vote by the FDA was closely watched by more than just sufferers of lupus. As the Wall Street Journal reported, “The pharmaceutical industry is watching the decision for hints on the FDA’s stance toward products for hard-to-treat diseases. Such products often target a relatively small pool of patients and can cost tens of thousands of dollars a year.”
Benlysta is clearly the most important drug in the pipelines of Human Genome Sciences, Inc. and GlaxoSmithKline PLC. With the FDA’s approval of this drug, the fortunes of both companies will, no doubt improve, perhaps dramatically. After the announcement of the vote, there were likely many high-fives in both board rooms.
There is much to be learned about the causes and the effects of lupus. Benlysta failed to pass preliminary tests by the FDA and the companies worked with the agency to design tests with never-used combinations of several disease activity measures. As a result, the FDA voted to approve the drug. The questions about the drug’s effectiveness among African-Americans and the general U.S. population are still to be answered. However, patients with this disease can take heart in the fact that some relief may, at last, be on the way.