New Lupus Drug: A Long Time Coming

FDA approves Benlysta to treat lupus

/ Author:  / Reviewed by: Joseph V. Madia, MD

It has been more than half a century since the FDA last approved any drugs to treat lupus. Now there's a new medication going to market.

The U.S. Food and Drug Administration today approved Benlysta (belimumab) to treat patients with active, autoantibody-positive lupus (systemic lupus erythematosus) who are receiving standard therapy, including corticosteroids, antimalarials, immunosuppressives and nonsteroidal anti-inflammatory drugs.

"Benlysta is the first new Lupus drug in 50 years."

Benlysta 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.

Prior to Benlysta, FDA last approved drugs to treat lupus, Plaquenil (hydroxychloroquine) and corticosteroids, in 1955. Aspirin was approved to treat lupus in 1948.

Lupus is a serious, potentially fatal, autoimmune disease that attacks healthy tissues and usually develops between ages 15 and 44. “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, MD, MPH, director of the Office of Drug Evaluation II in the FDA’s Center for Drug Evaluation and Research.

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.

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.

Those receiving Benlysta during clinical studies reported more deaths and serious infections compared with placebo. The drug should not be administered with live vaccines. The manufacturer is required to provide a Medication Guide to inform patients of the risks associated with Benlysta.

The most common side effects in the studies included nausea, diarrhea, and fever (pyrexia). Patients also commonly experienced infusion reactions, so pre-treatment with an antihistamine should be considered.

Lupus is an autoimmune disease that attacks multiple organ systems and connective tissues in the body, is incurable, but treatable, and most people with Lupus will live a normal lifespan. Close to 200,000 people in the United States have lupus, with women being affected nine times more than men. Women of Afro-Caribbean descent are affected three times more often than other groups. The initial symptoms of lupus are fever, joint pains and fatigue. About 30% of lupus patients have dermatologic symptoms, with 30%-50% getting the characteristic butterfly shaped rash on the face. Another very serious complication is end stage renal disease (ESRD), and kidney transplants are common. There is no single genetic cause for lupus, but many different genes have been identified as contributors to developing lupus. The most important genes for lupus development are located in the chromosome 6 HLA region. Lupus can also be drug induced by quinidine, phenytoin (Dilantin), hydralazine (Apresoline) and procainamide (Pronestyl), but is fortunately reversible. An ANA (antinuclear antibody) blood test can be used to diagnosis lupus. Drugs used to treat lupus are frequently DMARDS (disease modifying antirheumatic drugs) such as Humira, Rituxan, Remicade, Methotrexate and Enbrel; Steroids and other immunosuppressants help reduce symptoms as well, with examples being Belimumab and Atacicept. Painkillers are common and often necessary.

Human Genome Sciences Inc., based in Rockville, Md., developed Benlysta and will co-market the drug in the United States with GlaxoSmithKline of Philadelphia.

Reviewed by: 
Review Date: 
March 10, 2011