While the Food and Drug Administration advisers recommended approval of Benlysta yesterday, they raised enough concerns about safety that U.S. sales may be limited, said Yaron Werber, a Citigroup analyst in New York, in a note. Rockville, Maryland- based Human Genome slipped $1.37, or 5.3 percent, to $24.51 at 4 p.m. in Nasdaq Stock Market composite trading, and has fallen 20 percent so far this year.
Benlysta, which would be Human Genome’s first marketed product, works well enough to outweigh risks of suicide, infection and cancer, the panel said. Two of the 15 members of the panel voted against recommending approval at the meeting in Adelphi, Maryland.
The drug’s approval came with “very public criticism of its modest benefit and lackluster results” in U.S. patients, Weber said. “The label will likely have noticeable restrictions and thin efficacy is already drawing a mixed reception from rheumatologists.”
Citigroup and Bank of America Corp. each downgraded their rating of Human Genome in separate notes today.
5 Million People
Benlysta would be the first treatment approved since 1958 for lupus, an incurable condition that causes the immune system to attack healthy cells and affects about 5 million people worldwide. Human Genome and London-based Glaxo expect a decision as early as Dec. 9. If the drug is cleared, sales may surpass $2.1 billion by 2014, according to the average estimate of five analysts surveyed by Bloomberg.
The panel’s vote was “terrific for the drug” and “most of all, terrific for patients,” said Thomas Watkins, Human Genome’s chief executive officer, in an interview on CNBC today.
As for the safety concerns, “you have to put that all in context,” he said. “And I think that’s what the FDA panel did when it voted” to recommend approval. He declined to comment on the downgrades.
While the FDA isn’t required to follow the advisory panels’ recommendations, the agency usually does.
“The efficacy is mild, but there is a need for a drug even with mild efficacy, given the current state of therapies,” panel member Lenore Buckley, a professor of internal medicine and pediatrics at Virginia Commonwealth University in Richmond, said before the vote.
Glaxo advanced 29.5 pence, or 2.4 percent, to 1,243 pence at the close of London trading.
About 1.5 million Americans have lupus, which claimed the lives of novelist Flannery O’Connor and CBS television reporter Charles Kuralt. It’s most common in women ages 15 to 45 and three times more prevalent in black women than white women, according to the Department of Health and Human Services.
Benlysta, an intravenous drug also known as belimumab, inhibits the production of antibodies that attack healthy cells in lupus patients. The treatment, if approved next month, may become available in the first quarter of 2011, Thomas Watkins, Human Genome’s chief executive officer, said after the meeting.
While Benlysta is “almost certain to be approved,” the FDA may delay its decision up to three months beyond the Dec. 9 target date, Geoffrey Porges, an analyst at Sanford C. Bernstein & Co. in New York, said yesterday in a research note. The agency may need extra time to consider labeling requirements, post- marketing study obligations and risk-management strategies, Porges said.
FDA clearance of Benlysta may “usher in a new era of enlightenment” in lupus research, Sandra Raymond, president of the Washington-based Lupus Foundation of America, said during a public-comment session before the vote. Raymond was among more than 30 speakers who urged the panel to recommend approval of the drug.
Lupus symptoms range from mild to life-threatening, complicating the development of drugs and clinical trials, said Bonnie Bermas, director of the Brigham & Women’s Hospital Lupus Center in Boston.
“One patient may have a low white blood-cell count, fluid around their heart and skin rashes. Another patient could have arthritis and central nervous symptoms, and a third patient could have kidney disease,” Bermas said last month in a telephone interview. “They can all look like very different patients.”
Roche Holding AG and Biogen Idec Inc.’s Rituxan, approved for rheumatoid arthritis, leukemia and non-Hodgkin’s lymphoma, failed in a late-stage lupus trial last year. Teva Pharmaceutical Industries Ltd.’s Edratide, Genelabs Technologies Inc.’s Prestara and Riquent, developed by La Jolla Pharmaceutical Co. and BioMarin Pharmaceutical Inc. are among the proposed lupus treatments that also have fallen short.
The FDA is considering Benlysta based on a six-month priority review. While the agency typically takes at least 10 months to decide drug applications, it grants priority reviews to therapies that may provide major advances in treatment.
Benlysta’s risks of suicide, infection and cancer were greater than those in patients taking a placebo in clinical trials, Food and Drug Administration staff reviewers said Nov. 12 in a preliminary report. Data showing the drug was effective lacked “robustness,” the agency staff said.
Likely to Win
The drug is still likely to win approval because it met its primary goal of reducing patients’ symptoms in two late-stage clinical trials, Christopher J. Raymond, an analyst with Robert W. Baird & Co. in Chicago, said Nov. 12 in a telephone interview. The FDA’s recognition of an unmet medical need for more lupus therapies may outweigh agency staff’s concerns that the drug may not be safe or effective enough, he said.
Benlysta, if approved, would be the first treatment developed specifically for lupus. The steroid prednisone and some antimalarial drugs were already approved for different conditions when the FDA cleared them for use in lupus patients more than 50 years ago.
The chemotherapy drug cyclophosphamide and Roche’s CellCept, used in organ-transplant patients, are among medications prescribed for lupus patients without FDA approval. While FDA rules allow doctors to prescribe drugs for unapproved purposes, companies can’t market the treatments for those so- called off-label uses.
The long-term use of existing lupus treatments can have debilitating side effects that are worse than the disease itself, Sandra Raymond said, citing stroke, heart attacks, bone loss, hypertension, infertility and infections.
Benlysta may not work for all lupus patients, Brigham & Women’s Bermas said. Candidates may include people who have failed previous treatments or require high doses of prednisone to control their symptoms and want to lower the amount to prevent side effects, she said.
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