March 9, 2011 -- The FDA has approved Benlysta, the first new lupus treatment in 50 years.
An FDA advisory panel last November voted 13-2 in favor of approval. But the panel noted that Benlysta is no wonder drug. Overall, it offered a modest benefit. Only 30% of patients who took the drug in clinical trials saw a benefit.
And because the drug weakens the body's immune defenses, it comes with serious side effects. These include infections, cancers, depression, and suicide.
But the announcement comes as welcome news to patients and doctors frustrated by the limited treatment options available to lupus patients. And it comes even as scientists gain a new understanding of what lupus is, what goes wrong, and where researchers should look for new treatments.
What should lupus patients and their families know about Benlysta? WebMD consulted Eric L. Greidinger, MD, chief of rheumatism and immunology at the University of Miami Miller School of Medicine, FDA briefing documents, and the FDA approval announcement.
Why are new lupus drugs needed?
Officially known as systemic lupus erythematosus (SLE), lupus is an autoimmune disease. It's relatively common, affecting about one in 1,000 people. But some people with lupus have such mild disease they may never know they have it.
Others have relatively mild disease that can be controlled with current treatments. These include over-the-counter NSAIDs such as ibuprofen, corticosteroids such as prednisone, antimalaria drugs such as hydroxychloroquine, powerful immunosuppressants, and cancer chemotherapies. (Lupus is not caused by malaria and is not a cancer, but malarial drugs and chemotherapies suppress various manifestations of lupus).
Still other patients experience frequent lupus flare-ups and suffer devastating side effects from current treatments. And finally, there are patients with life-threatening lupus, at risk of major organ failure.
"In all those cases, the current drugs -- while not perfect -- provide a good series of choices," Greidinger says.
Patients with mild disease may not need treatment, or may be able to keep their symptoms under control with relatively safe antimalaria drugs.
But patients in the middle category are more difficult to treat, Greidinger says. They may not get relief from the safest lupus treatments. But stronger treatments, continued over time, may cause side effects that are worse than a patient's symptoms.
Which lupus patients might benefit most from Benlysta?
Many patients with mild-to-moderate lupus can't keep their flare-ups under control with antimalaria drugs.
"Their only real option at present is the use of corticosteroids like prednisone," Greidinger says. "But many of these patients have persistently active disease and thus may be exposed to substantial doses of steroids for months and years with a very high risk of serious side effects."
There's no proof that all, or even most of these patients will benefit from Benlysta. But many just might, says Greidinger, who refers to the drug by its generic name: belimumab. "The hope is belimumab may allow some of these patients who are using substantial doses of steroids to bring their disease under better control, so they have less lupus activity and be able to take lower doses of steroids."
That's what happened in clinical trials of Benlysta. On average, patients taking the drug were able to reduce the doses of prednisone they were taking.
In those studies, most of the benefit of Benlysta seemed to come from improvement of skin rash and ulcers in the nose and mouth.
"These symptoms can be prominent drivers of the disability patients have, and prominent factors driving their ongoing steroid use. Patients of that sort may be the most appropriate ones to begin trying this drug," Greidinger says.
Which patients are least likely to benefit from Benlysta?
Analysis of the data suggests that patients of African descent might not benefit from the drug -- but as Greidinger notes, there were too few such patients in the studies to know for sure.
What may be an issue for patients is that Benlysta is a biological treatment -- a man-made antibody -- that is expensive to produce. Most other such drugs carry a high price tag. However, reports in the financial press suggest that managed care organizations will cover Benlysta.
"Initially it is appropriate that not every rheumatologist rush out and put everyone of their patients on this drug, and not every patient should run out and ask for this drug," Greidinger says. "I think that as more experience develops with the drug, if the initial data is supported and supplemented by good clinical experiences, it may turn out to be cost-effective if it is preventing patients from getting serious complications of lupus, from getting side effects from prednisone therapy, and from being so disabled they cannot work."
Is Benlysta safe?
In clinical trials, there were more deaths and serious infections among lupus patients taking Benlysta than among those taking placebo plus standard therapy. People taking Benlysta cannot receive live vaccines.
The most common side effects in clinical trials were nausea, diarrhea, and fever. Patients also commonly experienced infusion reactions, so treatment with an antihistamine may be needed before each infusion.
A medication guide explaining all risks of Benlysta will be provided to all patients.