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FDA approves Yondelis for treatment of specific unresectable or metastatic soft tissue sarcomas
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The FDA approved the chemotherapeutic agent trabectedin to treat liposarcoma and leiomyosarcomas — soft tissue sarcomas that are difficult or impossible to remove surgically or that have metastasized — in patients who had previously been treated with anthracycline-based chemotherapy.
Trabectedin (Yondelis, Janssen), a synthetically produced anti-tumor agent derived originally from a sea squirt, works by binding to the DNA of cancer cells and disrupting their normal cell activity, which causes cell death, according to Janssen.
This marks the first treatment to be specifically approved for liposarcoma in the U.S. in 3 decades and could be viewed as a milestone for patients living with these diseases, which are rare and aggressive with limited approved treatment options. In 2014, there were an estimated 12,000 cases of soft tissue sarcomas diagnosed in the U.S., according to the National Cancer Institute.
“The treatment of advanced or metastatic soft tissue sarcoma represents a difficult challenge with few effective therapeutic choices available for patients,” Richard Pazdur, MD, director of the office of hematology and oncology products in the FDA’s Center for Drug Evaluation and Research, said. “[This] approval of Yondelis provides a treatment option for advanced or metastatic liposarcoma and leiomyosarcoma.”
When making its approval, the FDA relied on data from a phase 3 trial that measured its efficacy and safety in 518 patients with metastatic or recurrent liposarcoma or leiomyosarcoma.
A total of 345 patients were randomly assigned trabectedin, while the other 173 patients were randomly assigned an alternate chemotherapeutic agent, dacarbazine.
The patients in the trabectedin arm had a median PFS of 4.2 months compared with those in the dacarbazine group, whose median PFS was 1.5 months.
As for a safety profile, trabectedin’s most common side effects included fatigue, nausea, diarrhea, vomiting, dyspnea, peripheral edema, neutropenia, thrombocytopenia, anemia, headaches, decreased appetite, an increase in liver enzymes and a decrease in albumin, a blood protein.
The labeling for trabectedin contains a warning for the risk of neutropenic sepsis, hepatoxicity, rhabdomyolysis, cardiomyopathy, tissue necrosis and extravasation. Patients hypersensitive to the drug should not use it as part of their chemotherapy.
Additionally, pregnant women should neither take trabectedin, nor should they breastfeed while taking the drug. – by Anthony SanFilippo