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It can be a difficult disease to diagnose and a difficult disease to treat. It’s called lupus, and as many as 24,000 people in the United States are diagnosed with the disease each year. Scientists today are working on many fronts to understand the genetic underpinnings of the disease and to develop new and more targeted therapies to treat it.

What Is Lupus?

Lupus is a disease that can damage many parts of the body, including the joints, skin, kidneys, heart, lungs, blood vessels and brain. It is an autoimmune disease—an illness that occurs when the body mistakenly detects its own tissue as foreign and attacks itself, and it can be fatal in some severe cases. While people of all races can have the disease, African American women have a three-times higher number of new cases than white, non-Hispanic women. African American women tend to develop the disease at a younger age than white, non-Hispanic women and to develop more serious and life-threatening complications. It is also more common in women of Hispanic, Asian and Native American descent.

The underlying cause of lupus is not fully known, and there are many types of the disease. The most common form, called systemic lupus erythematosus, commonly causes mouth sores, rash, fatigue, joint pain and swelling, and affects the kidneys.

Lupus also is a chronic disease, meaning that treatment may help control it, but it never goes away, explains Nikolay Nikolov, M.D., a rheumatologist at the U.S. Food and Drug Administration. A person with lupus will have good periods and bad periods, he says, and a range of symptoms, which include extreme tiredness, pain or swelling in the joints, and headaches.

Who Is Affected?

The number of people living with systemic lupus in the United States is estimated to be approximately 200,000. According to the American College of Rheumatology, 10 times more women than men have lupus, and the disease often starts between the ages of 15 and 44.

What makes lupus so hard to diagnose? "People with lupus can have different problems with their immune systems," Nikolov says. And many of the symptoms that can occur in someone with lupus are non-specific and can also occur in other diseases, making it hard to nail down the diagnosis.

There is still an enormous need for better therapeutics. Scientists may be on the cusp of developing more refined therapies to help control symptoms and foster remission. The FDA’s Office of Women’s Health has funded several studies related to lupus and other autoimmune diseases in recent years. The FDA's Office of Minority Health and Health Equity also has funded a study on lupus.

According to RADM Richardae Araojo, FDA Associate Commissioner for Minority Health, "With racial and ethnic minority communities being disproportionately impacted by lupus, it is critical to advance research into therapeutic treatments and increase diversity in clinical research to help ensure that the medical products that are being developed work for the people they are intended to help."

Targeted Therapies

Treatment of lupus depends on the part of the body being affected by the disease and the severity of the problem. The FDA approved the first drug to treat lupus, aspirin, in 1948 and later approved corticosteroids, such as prednisone, which suppress the immune system and reduce inflammation. In 1955, the agency approved the antimalarial drug Plaquenil (hydroxychloroquine) which helps to relieve some lupus symptoms such as fatigue, rashes, joint pain or mouth sores.

"Part of what makes lupus research such a challenge is that the precise problem with the immune system is so different among patients and the disease manifestations are so variable." Nikolov says. New research is trying to zero in on what the best targets might be.

“The evolving understanding of the disease in recent years allowed the development of medicines that target the specific molecule or molecules in the immune system that may be causing the problem,” Nikolov says. “Older medicines tend to suppress the whole immune system, which can be an effective but an unnecessarily excessive measure that can be associated with many undesirable side effects.”

The FDA approved Benlysta (belimumab)—the first targeted therapy for lupus—in 2011 to treat systemic lupus in adults. The FDA later approved Benlysta to treat children with lupus down to 5 years of age. In December 2020, the FDA approved Benlysta as the first targeted therapy to treat adult lupus patients with kidney involvement—one of the more severe forms of this disease. Benlysta is designed to target a protein called B-lymphocyte stimulator, which may reduce the impact of abnormal cells thought to be a factor in the development of lupus. It is available in two forms--for injection directly into the vein or into the skin.

In July 2021, the FDA approved Saphnelo (anifrolumab-fnia) as the second targeted therapy to treat adults with systemic lupus. It is designed to target and reduce the impact of the Type 1 Interferon Receptor, which may be important in lupus. It is available in one form—for direct delivery into the vein.

Advances in the understanding and treatment of lupus over the last several decades have resulted in people with the disease living longer and better lives.

Despite these advances, however, there remain many people with lupus who need additional treatment options. The FDA remains committed to working with researchers, drug developers, and patients to help make new treatments a reality.