FDA approves first new drug for lupus in 56 years

April 1, 2011

FDA approved belimumab (Benlysta, Human Genome Sciences and GlaxoSmithKline) for the treatment of adult patients with active, autoantibody-positive systemic lupus erythematosus who are receiving standard therapy.

FDA approved belimumab (Benlysta, Human Genome Sciences and GlaxoSmithKline) for the treatment of adult patients with active, autoantibody-positive systemic lupus erythematosus (SLE) who are receiving standard therapy.

Belimumab is the first in a new class of drugs called B-lymphocyte stimulator (BLyS)-specific inhibitors. BLyS is a naturally occurring protein that was discovered by HGS in 1996. Belimumab is made available as a lyophilized powder in single-use vials for intravenous infusion only and must be reconstituted and diluted by a healthcare professional prior to administration.

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

“We expect to have this novel therapy available to physicians and patients within about 2 weeks, and our entire organization looks forward to the positive impact we hope this new therapy will have for patients with systemic lupus,” H. Thomas Watkins, president and CEO, HGS, said in a company statement.

Two clinical studies involving 1,684 patients with lupus demonstrated the safety and effectiveness of belimumab. In the studies patients diagnosed with active lupus were randomly assigned to belimumab 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 belimumab and standard therapies experienced less disease activity than those who received 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 2 studies did not appear to respond to treatment with belimumab. 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 belimumab for this subgroup of lupus patients.

Those receiving belimumab 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 belimumab.

The most common side effects in the studies included nausea, diarrhea, and fever (pyrexia). Patients also commonly experienced infusion reactions, so pretreatment with an antihistamine should be considered. “There are a number of pioneering biotechnology and pharmaceutical companies, involved in the research and development of new treatments for lupus, and our hope is that [the approval] will further stimulate additional companies to invest in new therapies for lupus,” said Sandra C. Raymond, president and CEO of the Lupus Foundation of America (LFA), Washington, D.C., in a company statement.

To build on this momentum and encourage the development of new treatments, the LFA has launched new initiatives that help to strengthen clinical trials. These programs include the launch of a Web-based program designed to train clinical investigators on the instruments used in trials. The LFA recently implemented the LFA Lupus Research Registry that enables individuals to be notified about new clinical trials in their geographic area. The Registry is part of the LFA’s Center for Clinical Trials Education.

“The LFA also is partnering with key stakeholders from industry, government, and the scientific community to evaluate data from previous lupus clinical trials with the goal to improve the design of future studies,” Raymond added.

"Although belimumab [Benlysta] represents a welcome addition to the armamentarium of drugs used to treat a relatively rare condition, it will be interesting to see the impact of this new agent," said Robert T. Taketomo, PharmD, MBA, president/CEO and chairman of the board, Ventegra, LLC, a Glendale, Calif.-based managed care contracting services organization representing payers, providers, PBMs, and government programs, told Formulary. "Like drugs used to treat many other incurable diseases, the financial success of belimumab will hinge upon how the balance between therapeutic effectiveness and hope plays out between providers of care and patients."