Drug approval means that patients who have this rare cancer will now have access to the first new treatment for soft tissue sarcoma in decades. The rare cancer begins in the muscle, fat and other soft tissues and Votrient works by interfering with the growth of new blood vessels that solid tumor need to grow.
Soft tissue carcinoma has many subtypes and occurs in an estimated 10,000 cases annually in the United States. Votrient received orphan drug status for this indication, a designation reserved for rare diseases affecting fewer than 200,000 patients. The drug approval does not include adipocytic soft tissue sarcoma and gastrointestinal tumors.
“Drug development for sarcomas has been especially challenging because of the limited number of patients and multiple subtypes of sarcomas,” Dr. Richard Pazdur, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research said in a statement.
Britain-based GSK, which has its U.S. headquarters in Research Triangle Park, North Carolina, first received approval for Votrient in 2009 to treat advanced kidney cancer. Votrient’s approval to treat soft tissue carcinoma patients who have already been treated with chemotherapy follows a positive vote last month from an FDA advisory panel. But the pill does come with a black box warning that notes the risk of potentially fatal liver damage. The warning advises that patients on Votrient be monitored for liver function; if liver function declines, treatment should be stopped.