GlaxoSmithKline Plc won the backing of U.S. advisers for a drug to treat a rare and often deadly form of cancer.
A Food and Drug Administration advisory panel voted 11-2 today in Silver Spring, Maryland, that the benefits outweigh the risks including liver toxicity and heart dysfunction of an expanded approval for Votrient. The agency isn’t required to follow the panel’s recommendation. The advisers will vote later today on a similar treatment developed by Merck & Co. and Ariad Pharmaceuticals Inc.
Glaxo is seeking U.S. clearance for the medicine as the first targeted treatment for a cancer that invades soft tissue such as muscle, tendons and fat outside the stomach and intestines. Chemotherapy is now used for these tumors. New drugs are needed even if the benefit isn’t as great as hoped, panel members said.
“It’s a little step but it’s a little step in a field that’s had nothing,” said panel member Lee Helman, scientific director for clinical research at the National Cancer Institute’s center for cancer research, during the meeting. “This is a drug I would like to have in my armamentarium.”
Votrient was approved in 2009 to treat advanced renal cell carcinoma. Glaxo rose less than 1 percent to 1,433 pence at 3:55 p.m. London time.
FDA staff questioned the drug’s benefit in a report March 16 because the treatment didn’t prolong patients’ lives even though it kept sarcoma from spreading in soft tissue. The median survival of patients diagnosed with soft-tissue sarcoma that is spreading is about a year, according to the agency documents. The regulator is set to decide on London-based Glaxo’s drug by May 6.
About 10,000 cases of soft-tissue sarcoma are diagnosed each year, Richard Riedel, an oncologist and assistant professor at the Duke University School of Medicine in Durham, North Carolina, said in a telephone interview.
There are about 50 types of soft-tissue sarcoma, which are different from carcinomas that arise from cells, according to the American Cancer Society. Most sarcomas are in the arms and legs or internal organs, according to the Bethesda, Maryland-based National Cancer Institute.
Chemotherapy is effective at shrinking the tumor in 20 percent to 25 percent of patients, said Scott Okuno, an oncologist at the Mayo Clinic in Rochester, Minnesota. Once tumors have spread, a minority of patients are cured, he said in a telephone interview.
In rare cases, tumors develop in the stomach and small intestine. Two existing drugs are approved for these gastrointestinal sarcomas: Pfizer Inc.’s Sutent and Novartis AG’s Gleevec.
Votrient, chemically known as pazopanib, enabled patients to live a median 4.6 months, or 3.1 months longer than placebo, while the disease stopped advancing, according to a Glaxo statement. The final-phase trial studied 369 patients, 246 on the drug, with certain metastatic soft tissue sarcomas that didn’t respond to chemotherapy.
Median survival was 12.6 months for patients taking Votrient and 10.7 months for those on placebo.
Of the patients on Votrient, 98 experienced a serious adverse event compared with 29 on placebo, according to the FDA staff report. Forty-seven patients on Votrient discontinued the treatment, compared with six on placebo, because of side effects such as signs of a liver disorder, shortness of breath, high blood pressure and a blockage in the main artery of the lung.
The trial excluded patients with gastrointestinal tumors.