TUESDAY, Nov. 16 (HealthDay News) -- U.S. Food and Drug
Administration advisers voted 13-2 Tuesday to recommend approval of
the first new drug in more than 50 years to treat the autoimmune
The drug, belimumab (Benlysta), is a so-called monoclonal
antibody drug developed to treat patients who suffer from systemic
Lupus is an autoimmune disease in which the body produces
antibodies that attack and destroy healthy tissue. This can damage
joints, skin, blood vessels and organs. Benlysta works by
inhibiting the production of these antibodies.
While the FDA is not bound by its advisory committees'
recommendations, it usually follows them.
Benlysta is made by Human Genome Sciences and
In a report submitted to the advisory committee before the vote,
the FDA had expressed some concerns about the efficacy and safety
of the drug.
But other experts expressed optimism about the drug's worth.
"I really do think the drug is efficacious," said Sandra C. Raymond, president & CEO of the Lupus Foundation of America, who attended the advisory panel meeting. "What I'm hearing in terms of efficacy is pretty strong."
In a statement commenting on the panel's vote, Margaret Dowd,
president and CEO of the Lupus Research Institute, said that "we
couldn't be more pleased. It's a historic day for the lupus
The FDA said it wanted the advisory panel to look at both the
efficacy data, which according to the agency was modest, and the
safety data, which the agency believed raised some serious issues.
Specifically, the drug has been linked to depression and
Raymond noted, however, that many patients with lupus suffer
from depression. And many patients in clinical trials testing the
drug were also taking antidepressants, she added.
The advisory committee actually took three votes Tuesday. The
other two votes endorsed the safety of the drug (14-1 in favor) and
its effectiveness (10 to five in favor).
The agency said it based its concerns on the results of two
phase 3 trials that compared Benlysta with a placebo.
"The risks associated with belimumab include infection, malignancy, suicidality and overall number of deaths with a numerical imbalance that favored placebo treatment over belimumab treatment," according to the FDA paper filed before the vote.
Safety is important, Raymond said, but the drugs used to treat
lupus now carry some significant side effects, too. "The safety of
some of our medications is not good," she said. "So what are we
comparing it to?"
The current treatments include chemotherapy and steroids, which
can leave the door open to infections, cancers and other unwanted
consequences, Raymond said.
Dr. Joan Merrill, head of clinical pharmacology at the Oklahoma
Medical Research Foundation and medical director of the Lupus
Foundation of America, added that "the safety data on Benlysta
looks better than we usually see with biologics."
"We have to keep in mind that there is a natural ceiling on the percentage of patients who can respond to any targeted treatment. We do not yet know exactly how good Benlysta is," Merrill said.
What is known is that Benlysta was shown to be statistically
superior to a placebo in two phase 3 trials, she said.
"I hope that the FDA will be able to approve it, after they complete the serious deliberations that they must make on all the data," Merrill said.
Another expert agreed.
"I am hoping the advisory committee will recommend approval of Benlysta, because it has been associated with statistically significant improvement in the response to treatment in two well-designed randomized, prospective, placebo-controlled trials studying different populations of people with systemic lupus," said Dr. Bevra H. Hahn, chief of rheumatology at UCLA's David Geffen School of Medicine.
"Very important are the observations that fatigue [a major complaint of almost everyone with lupus] and physical function improved significantly," she added.
To learn more about lupus, visit the
Lupus Foundation of America.
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