When Benlysta was considered by an FDA advisory committee last November, its members were asked whether the drug had demonstrated “substantial evidence of efficacy in reducing disease activity” among the 1,300 patients who had received the drug in clinical trials. The panel responded positively, but not exactly enthusiastically — with a 10-5 vote, a relatively lukewarm endorsement compared with other approved drugs.
Although there were patient anecdotes — such as Janice Fitzgibbon’s — about the benefits of Benlysta, the drug’s impact was not overwhelming. It was only after Human Genome Sciences, the drug’s maker, narrowed the type of patients included in its study that Benlysta showed any statistically relevant effects at all. It should be noted that the patients participating in the study were already being treated with two other drugs, which set a high hurdle for Benlysta to show additional benefits.
Yet the drug appeared to be relatively safe, and until Benlysta, lupus drugs had all been created to treat other diseases. So when the committee was asked, in a second vote, whether the FDA should license Benlysta, three of the naysayers switched sides, for a 13-2 vote.
The FDA approved marketing of the drug in March, and it went on sale in April.
Because the drug costs about $35,000 a year per patient, it’s unclear how widely it will be used, doctors say, and whether poorer patients, or those on Medicaid, will have access.
In the short time the drug has been on the market, HGS vice president Barry A. Labinger said, insurance companies have shown a willingness to cover it, and the company has provided subsidies for under- and uninsured patients. He declined to provide sales figures for the drug.
Meanwhile, the company has begun enlisting African American patients for a separate trial requested by the FDA to study whether the drug is effective in that group. One of the trials conducted before approval suggested that the drug provided no benefit to African Americans, but that result may have been a statistical fluke.
— Arthur Allen