FDA expresses concerns about lupus drug's effectiveness

By Steven Overly
Monday, November 15, 2010

Rockville-based Human Genome Sciences got a taste last week of the questions it may receive when the biotechnology firm goes before a Food and Drug Administration panel on Tuesday seeking an endorsement for its new drug to treat lupus.

In briefing documents posted online Friday, the FDA expressed concerns over whether the drug, called Benlysta, is effective over the long term and among specific patient groups. The agency also questioned whether potential adverse effects of treatment, such as infections or depression, trump the benefits.

"Various analyses of the data raise questions about the robustness of the efficacy findings that warrant discussion" at Tuesday's meeting, the agency said. The committee's vote on whether to support the drug could impact whether the FDA grants final approval next month.

Shares of Human Genome Sciences closed at $23.60 on Friday, down nearly 11 percent, or $2.88, compared with the day before.

Among the concerns noted in the review:

-- African Americans showed a negative response to the drug compared with other racial groups.

-- The benefits experienced by patients in the United States and Canada were less than those in Latin America.

-- Patients using the drug showed fewer disease symptoms during the first year of treatment, but after 76 weeks did not fare much better than those on a placebo.

-- Patients using Benlysta, an immunosuppressant, were at a higher risk of infection and suicide.

Human Genome Sciences declined to comment in advance of the committee meeting.

Benlysta would be the first drug for lupus approved in more than 50 years, and patient advocates said it still presents an attractive alternative to current treatments. Systemic lupus is a complicated autoimmune disease in which the body attacks its organs.

"None of the drugs are going to perfect. Benlysta is not going to be a miracle cure for all people with lupus. It is going to be helpful to some people with lupus and then that door will be open and it will spur research," said Sandra Raymond, president and chief executive of the Lupus Foundation of America.

Joseph Schwartz, a biotechnology analyst for Leerink Swann, said he did not think the findings would prevent the drug from reaching patients. But he said the concerns could delay the final approval as regulators and drugmakers determine a strategy to best inform patients of the risks.

Due to the unmet medical need and a potential patient pool of 5 million people worldwide, analysts have previously predicted that Benlysta's approval could mean billion-dollar revenues for Human Genome Sciences and GlaxoSmithKline, its partner on the drug. That would add the Rockville company to the list of biotech darlings, including MedImmune and Qiagen, along Maryland's Interstate 270.

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