ROCKVILLE, Md.—Despite the U.S. Food and Drug Administration (FDA) raising questions about the efficacy and safety of belimumab—trade name Benlysta, the first lupus drug in more than 50 years to be approved by the FDA—a leading lupus expert says approval of the drug is "a step in the right direction" for patients who suffer from the difficult-to-treat chronic autoimmune disorder.
"The FDA, rightly so, has expressed concerns," says Dr. Joan T. Merrill, head of the Clinical Pharmacology Research Program at the Oklahoma Medical Research Foundation (OMRF), a leading center for lupus research in the United States. "Benlysta may work for some, but not all, patients with lupus. We still don't 100 percent know who those patients are. Also, we won't know for 20 years about all of the possible side effects."
Merrill is also the Lupus Foundation of America's (LFA) first medical director, and in that capacity, she assists the organization's board of directors and professional staff to implement expansion of the organization's programs of biomedical research, professional education and public policy. The LFA called March 9, the day Benlysta received approval, "a historic day for the millions of people with lupus and their families around the world," and "the beginning of a new era of improved diagnosis, prevention and treatment for the disease."
"Until about 10 years ago, lupus was underdiagnosed and misunderstood," Merrill says. "There wasn't much interest in lupus research among pharma companies."
Benlysta's origins go back about that far, as Human Genome Sciences (HGS) in 1996 discovered B-lymphocyte stimulator, or BLyS, is overproduced in human systemic lupus erythematosus (SLE). Considerable evidence has shown that in lupus, rheumatoid arthritis and certain other autoimmune diseases, elevated levels of BLyS are believed to contribute to the production of autoantibodies that attack and destroy the body's own healthy tissues. The result is an overactive immune response that attacks otherwise healthy cells and tissue, wreaking havoc on the joints, skin and other organs.
Benlysta, which HGS co-developed with GlaxoSmithKline, is a human monoclonal antibody drug that inhibits the biological activity of BLyS. Belimumab does not bind B cells directly, but by binding BLyS, belimumab inhibits the survival of B cells, including autoreactive B cells, and reduces the differentiation of B cells into immunoglobulin-producing plasma cells.
The FDA has approved Benlysta for the treatment of adult patients with active SLE who are receiving standard therapy.
The drug holds great promise for the estimated 1.5 million people in the United States—most of whom are women—who suffer from lupus, says Merrill.
"Benlysta has a very nice safety profile and that its benefits clearly outweigh its risks," she says. "Considering that the old-fashioned approach and standard of care has been to prescribe steroids to keep the disease under control—which unfortunately invokes side effects that make this intolerable for a lot of patients—this is a step in the right direction."
The drug's efficacy has not been evaluated in patients with severe active lupus nephritis or severe active central nervous system lupus, and has not been studied in combination with other biologics or intravenous cyclophosphamide.
The FDA has raised concerns about Benlysta in certain patient groups. Specifically, regulators are raising questions about the efficacy and safety of the drug in black patients with lupus, as only a small number of these patients were included in clinical trials. The FDA is also concerned about Benlysta's efficacy over time as well as its safety for pediatric or pregnant patients.
But despite these concerns, and the fact that no two lupus patients are alike—thus rendering a "one drug fits all" approach impossible, Merrill is hopeful that the approval of Benlysta is just a first step in drug companies realizing what an underserved market lupus is.
"Now that Benlysta has been approved, these researchers can go to their executives for the first time in 15 years and confidently encourage them to invest in lupus treatments," she says. "There's a light at the end of the tunnel."
"The FDA, rightly so, has expressed concerns," says Dr. Joan T. Merrill, head of the Clinical Pharmacology Research Program at the Oklahoma Medical Research Foundation (OMRF), a leading center for lupus research in the United States. "Benlysta may work for some, but not all, patients with lupus. We still don't 100 percent know who those patients are. Also, we won't know for 20 years about all of the possible side effects."
Merrill is also the Lupus Foundation of America's (LFA) first medical director, and in that capacity, she assists the organization's board of directors and professional staff to implement expansion of the organization's programs of biomedical research, professional education and public policy. The LFA called March 9, the day Benlysta received approval, "a historic day for the millions of people with lupus and their families around the world," and "the beginning of a new era of improved diagnosis, prevention and treatment for the disease."
"Until about 10 years ago, lupus was underdiagnosed and misunderstood," Merrill says. "There wasn't much interest in lupus research among pharma companies."
Benlysta's origins go back about that far, as Human Genome Sciences (HGS) in 1996 discovered B-lymphocyte stimulator, or BLyS, is overproduced in human systemic lupus erythematosus (SLE). Considerable evidence has shown that in lupus, rheumatoid arthritis and certain other autoimmune diseases, elevated levels of BLyS are believed to contribute to the production of autoantibodies that attack and destroy the body's own healthy tissues. The result is an overactive immune response that attacks otherwise healthy cells and tissue, wreaking havoc on the joints, skin and other organs.
Benlysta, which HGS co-developed with GlaxoSmithKline, is a human monoclonal antibody drug that inhibits the biological activity of BLyS. Belimumab does not bind B cells directly, but by binding BLyS, belimumab inhibits the survival of B cells, including autoreactive B cells, and reduces the differentiation of B cells into immunoglobulin-producing plasma cells.
The FDA has approved Benlysta for the treatment of adult patients with active SLE who are receiving standard therapy.
The drug holds great promise for the estimated 1.5 million people in the United States—most of whom are women—who suffer from lupus, says Merrill.
"Benlysta has a very nice safety profile and that its benefits clearly outweigh its risks," she says. "Considering that the old-fashioned approach and standard of care has been to prescribe steroids to keep the disease under control—which unfortunately invokes side effects that make this intolerable for a lot of patients—this is a step in the right direction."
The drug's efficacy has not been evaluated in patients with severe active lupus nephritis or severe active central nervous system lupus, and has not been studied in combination with other biologics or intravenous cyclophosphamide.
The FDA has raised concerns about Benlysta in certain patient groups. Specifically, regulators are raising questions about the efficacy and safety of the drug in black patients with lupus, as only a small number of these patients were included in clinical trials. The FDA is also concerned about Benlysta's efficacy over time as well as its safety for pediatric or pregnant patients.
But despite these concerns, and the fact that no two lupus patients are alike—thus rendering a "one drug fits all" approach impossible, Merrill is hopeful that the approval of Benlysta is just a first step in drug companies realizing what an underserved market lupus is.
"Now that Benlysta has been approved, these researchers can go to their executives for the first time in 15 years and confidently encourage them to invest in lupus treatments," she says. "There's a light at the end of the tunnel."
