The U.S. FDA has finally given the green light to BENLYSTA,  a novel therapy developed by Human Genome Sciences and its partner GlaxoSmithKlin for the treatment of lupus, a historical move as the  first approval of new treatment for such a condition in over a half-century.

The U.S. regulator approved Benlysta (belimumab) for the treatment of adults with active, autoantibody-positive systemic lupus erythematosus (SLE) who are receiving standard therapy, including corticosteroid, anti-malarials, immunosuppressives and nonsteroidal anti-inflammatory drugs.
The FDA’s decision followed the recommendation of an advisory committee voting 13 to 2 in favor of the approval of Benlysta for such an indication last November, despite reservations of many on the committee that the drug was only marginally effective.

The autoimmune disease Lupus causes a range of symptoms including arthritis, kidney damage, chest pain, skin rashes, severe fatigue and other problems. The organ damage can be fatal. It is estimated that 322,000 Americans either definitely or probably had SLE, the most common form and the one for which Benlysta would be approved. According to the Lupus Foundation of America, an advocacy group, about 1.5 million Americans have some form of lupus. Presently there is no cure for lupus, but the condition can be controlled using immune-suppressing drugs like steroids.

The approval of Benlysta follows a series of setbacks of other companies trying to develop new drugs for lupus. FDA’s decision could encourage other pharmaceutical companies to pursue the treatment of the disease.

Dr. Curtis Rosebraugh, head of the FDA office that reviewed the drug, said, “Benlysta, when used with existing therapies, may be an important new treatment approach …to help manage symptoms”.

Benlysta reduces the disease’s level of activity by inhibiting a bodily protein called the B-lymphocyte stimulator, which at elevated levels can contribute to the creation of autoantibodies. Autoantibodies are the cells that attack otherwise healthy tissue, such as the skin, kidneys, heart and joints.

“We and GSK are honored to have the opportunity, with the approval of FDA, to bring BENLYSTA forward in the United States as the first new drug for systemic lupus in more than 50 years,” commented H. Thomas Watkins, President and Chief Executive Officer, HGS. “We expect to have this novel therapy available to physicians and patients within about two weeks, and our entire organization looks forward to the positive impact we hope this new therapy will have for patients with systemic lupus.”

The FDA noted that African-American patients and those of African heritage participating in two clinical trials involving 1,684 lupus patients did not appear to respond to treatment with Benlysta. The studies “lacked sufficient numbers to establish a definite conclusion”.  FDA has required the company to conduct an additional post-approval study in African-American patients, and HGS agreed to do so.

It is also noted that more deaths and serious infections were reported for patients receiving Benlysta during clinical trials compared with placebo. The drug should not be administered with live vaccines and the manufacturer is required to provide a medication guide to inform patients of the risks associated with the treatment.

The companies have not yet announced the price of Benlysta, but analysts expect it to be about $35,000 a year. Analysts estimated that  Benlysta annual global sales may top $3 billion in 2015.