FDA Approves First Lupus Drug in 50 Years

March 9, 2011— -- The U.S. Food and Drug Administration on Wednesday approved Benlysta, the first lupus drug in half a century.

The drug, made by Human Genome Sciences Inc., is designed to lessen the severity of lupus, a little-understood disease in which the body's immune system attacks tissues and organs, producing skin rashes, joint pain, chronic fatigue and in severe cases, heart and kidney complications.

Even though Benlysta has been shown to be only moderately successful in decreasing disease markers (an FDA advisory committee expressed some trepidation about its effectiveness last November), the dearth of other effective treatments makes Benlysta the hope of many a lupus researcher and patient.

"We still have a long way to go in understanding and treating lupus, but it's important to approve this right now. It really is the first drug for lupus to meet clinical endpoints [in decades]," says Dr. Betty Diamond, a lupus researcher who heads the Center for Autoimmune and Musculoskeletal Diseases at the Feinstein Institute.

Current standard of care for lupus includes high-dose steroids and anti-malaria medication to hamper the body's immune system. These medications can cause bone deterioration, infection, muscle weakness, ulcers and more, which, compounded with the symptoms of the disease itself, greatly hinder quality of life.

For Kathleen Nolan, 43, a registered nurse, currently available lupus treatments left her at the end of her rope. She still experienced severe fatigue and terrible joint pain that made it impossible for this registered nurse to get through the day without lying down.

"I was so frustrated. My doctor told me there was nothing more they could do for me," Nolan says. That was when she was referred to the Feinstein Institute in Manhasset, N.Y., where she was enrolled in a trial for the Benlysta.

Long-Awaited New Hope for Lupus

Although she saw her condition improve within a few months, it was impossible to know whether she was receiving Benlysta in that first year because she was in a double-blind, placebo-controlled trial, and may have been taking a placebo. But after that first year, promising feedback from other Benlysta trials led researchers to switch all participants to various doses of Benlysta.

"I'm able to exercise now. I still have some days that are better than others, but I can make it through the day without lying down. I can do housework again. This drug is such a major thing for us lupus patients," says Nolan, who has battled the disease for 15 years. "It gives us some hope."

FDA's Wavering on Benlysta's Safety

When Benlysta was brought before an FDA advisory panel last November, the members raised questions regarding the drug's efficacy and safety. For one, Benlysta doesn't seem to work for African-American patients.

"It's not clear what the drug's effects will be in African-Americans," says Dr. Howard Belmont, a lupus expert at New York University Langone Medical Center. African-Americans tend to have a more aggressive form of the disease, and are resistant to treatment in general, he said, which may explain the clinical trials less-than stellar results for this population.

Benlysta's noted side effects included infection, depression and suicide, all of which have delayed its approval.

Dr. Cynthia Aranow, co-director of the program for autoimmune diseases and clinical research at the Feinstein Institute, who was involved in the Benlysta trials, calls the safety profile for the drug "excellent," especially in light of the current standard of care for lupus. Since Benlysta seems to allow for reduced doses of steroids, which carry numerous negative side effects, she said it might offer "a spectacular new option for lupus treatment."

Limited Application for Promising New Drug

But even now that it has been approved, Benlysta will likely work in tandem with other lupus medications, and since Benlysta was not tested on patients with severe lupus in which the kidneys are heavily affected, it will most likely be prescribed only to those with mild to moderate disease.

"It didn't work for all patients, but for those for whom it did, it did improve quality of life in treatment of what we call mild-moderate lupus," Belmont says.

Whether or not Benlysta will become the groundbreaking new treatment that many hope for, lupus experts were eager to see it approved.

"It's a long way from being a perfect drug, but I'll be interested to see if it can hold up as a usable drug," says Diamond.