Nov. 16, 2010 -- At the end of a day of often emotional testimony, an FDA advisory panel overwhelmingly voted to recommend the approval of a new drug for the treatment of systemic lupus.
If the FDA follows the panel’s advice, the drug, belimumab, will be the first drug approved in more than 50 years for the chronic and debilitating autoimmune disease.
Despite the 13-2 vote in favor of approval, no member of the Arthritis Advisory Committee considered belimumab -- to be marketed as Benlysta -- a wonder drug.
“It has a weak effect, but they made the case,” panelist Matthew Liang, MD, MPH, a professor of medicine at Harvard, said of the data supplied by the drug’s manufacturer, Human Genome Sciences.
Systemic lupus erythematosus (SLE) causes the body’s immune system to attack healthy cells and tissues. Over time, it can damage the kidneys, heart, lungs, and other organs. Among its most common symptoms are extreme fatigue and swollen, painful joints.
Ninety percent of those with lupus are women, who are most often diagnosed during childbearing years. The CDC estimates that more than a million people in the U.S. may have the disease.
Several of the panelists expressed concern that the supporting studies submitted by the drugmaker demonstrated that the drug did not appear to help African-Americans, a population with a high risk for developing the disease.
“The lack of efficacy in African-Americans and people of African heritage needs to be studied,” said Christy Sandborg, MD, chief of pediatric rheumatology at Stanford University School of Medicine.
Overall, only 30% of those taking the drug benefited from it, one of the reasons for the panelists’ lukewarm response. That response, however, was not shared by the 30 members of the public who addressed them during the hearing, many of them choking back tears as they spoke.
Erica Corcoran, a participant in one of the studies, was a junior in college when she was diagnosed with lupus in 2004. The disease effectively ended life as she had known it. Unable to continue school or maintain any social life, her thoughts turned occasionally to suicide, she told the panel, until she began taking belimumab.
“It has been a wonder drug,” she said. “Since I have been on it, I have run a marathon and finished graduate school.”
Many others talked of the devastating effects of the disease, as well as the devastating side effects of the medications commonly prescribed to treat it.
According to Michelle Petri, MD, a professor of rheumatology at Johns Hopkins University and director of the Hopkins Lupus Cohort, corticosteroids such as prednisone, which are commonly prescribed for lupus, do more organ damage over the long term than the disease itself.
“Over 90% of my SLE patients remain on steroids,” said Petri, who spoke on behalf of HGS. “And [steroids] increase permanent organ damage.”
Belimumab, by comparison, comes with few safety concerns. In fact, the new drug may allow patients to reduce the amount of steroids that they take.
“It looks remarkably good compared to the other drugs that we are using,” said panelist R. John Looney, MD, a professor of medicine at the University of Rochester School of Medicine and Dentistry. “It seems like it is going to be significant in terms of what we are going to be using [with our patients].”