Glaxo, Human Genome Lupus Medicine May Not Be Safe, FDA Staff Report Says
By Molly Peterson - Nov 12, 2010
GlaxoSmithKline Plc and Human Genome Sciences Inc.’s experimental lupus drug Benlysta may not be safe, according to U.S. regulators weighing approval of the first new treatment for the autoimmune disorder in more than 50 years. Human Genome shares plunged 11 percent.
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 said today in a preliminary review on the agency’s website. Data showing the drug was effective lacked “robustness,” the agency staff said.
Outside advisers to the FDA will meet Nov. 16 to assess the medicine’s effectiveness and safety, and recommend whether it should be approved for sale. Benlysta would be the first treatment approved by the FDA since 1958 for lupus, a chronic condition that causes the immune system to attack healthy cells and affects about 5 million people worldwide.
While high doses of Benlysta met primary goals of two trials, there was a “lack of demonstrated efficacy” in black patients in the U.S. and an “inconsistent efficacy trend across different geographical regions of the world,” the agency staff said.
The FDA’s recognition of an unmet medical need for more lupus therapies may outweigh agency staff reviewers’ safety and efficacy concerns, Christopher J. Raymond, an analyst with Robert W. Baird & Co. in Chicago, said in a telephone interview.
‘Benign Safety Profile’
“If there’s one surprise, it’s that the FDA seems a little more concerned about the risk-benefit profile” than investors may have expected, said Raymond, who has an “outperform” rating on Human Genome. “Still, the drug has a relatively benign safety profile” and the advisory panel review of the drug “ought to go smoothly,” he said.
Human Genome, based in Rockville, Maryland, lost $2.88, or 11 percent, to $23.60 at 4 p.m. New York time in Nasdaq Stock Market composite trading, the biggest single-day decline about 19 months. Glaxo’s American depositary receipts fell 13 cents, or less than 1 percent, to $39.62 in New York Stock Exchange composite trading. Each receipt equals two ordinary shares of London-based Glaxo.
The companies expect a decision on approval by Dec. 9, and 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 concerns raised by FDA staff are part of a report that has positive overall findings, said Michael Yee, an analyst at RBC Capital Markets in San Francisco.
“None of it is particularly surprising,” Yee said of the report today in a telephone interview. The FDA may approve the drug without calling for additional trials, he said.
Fatal Disease
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 inhibits the production of antibodies that attack healthy cells in lupus patients.
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.
Roche Holding AG and Biogen Idec Inc.’s Rituxan, approved for rheumatoid arthritis, leukemia and non-Hodgkin’s lymphoma, failed a late-stage lupus trial last year. Other drugs that have fallen short include Teva Pharmaceutical Industries Ltd.’s Edratide, Genelabs Technologies Inc.’s Prestara and Riquent, developed by La Jolla Pharmaceutical Co. and BioMarin Pharmaceutical Inc.
Six-Month Review
The FDA is considering Benlysta based on a six-month priority review. While the agency typically takes at least 10 months to rule on drug applications, it grants priority reviews to therapies that may provide major advances in treatment.
Benlysta, if approved, would be the first drug developed specifically for lupus, said Sandra Raymond, president of the Washington-based Lupus Foundation of America. 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, an immunosuppressant used in organ-transplant patients, are among medications commonly 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.
Existing Treatments
The long-term use of existing lupus treatments can have debilitating side effects that, for many patients, 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.
To contact the reporter on this story: Molly Peterson in Washington at mpeterson9@bloomberg.net
To contact the editor responsible for this story: Reg Gale at rgale5@bloomberg.net. |
