FDA Approves Trabectedin for Certain Types of Advanced Soft Tissue Sarcoma

Approval concerns treatment of patients with unresectable and advanced liposarcoma and leiomyosarcoma

On 23 October, 2015 the US Food and Drug Administration (FDA) approved trabectedin (Yondelis) for the treatment of two types of soft tissue sarcomas (STS) – liposarcoma and leiomyosarcoma – that cannot be removed by surgery (unresectable) or is advanced (metastatic). This treatment is approved for patients who previously received chemotherapy that contained anthracycline.

Liposarcoma and leiomyosarcoma are specific types of STS that occur in fat cells (liposarcoma) or smooth muscle cells (leiomyosarcoma). STS can form almost anywhere in the body, but is most common in the head, neck, arms, legs, trunk and abdomen.

The effectiveness and safety of Yondelis were demonstrated in 518 clinical trial participants with metastatic or recurrent leiomyosarcoma or liposarcoma. Participants were randomly assigned to receive either Yondelis (345 patients) or dacarbazine (173 patients), another chemotherapy drug.

Participants who received Yondelis experienced a delay in the growth of their tumour (progression-free survival), which occurred on average about 4.2 months after starting treatment, compared to participants assigned to dacarbazine, whose disease progressed an average of 1.5 months after starting treatment.

The most common side effects among participants who received Yondelis were nausea, fatigue, vomiting, diarrhoea, constipation, decreased appetite, dyspnoea, headache, peripheral oedema, neutropaenia, thrombocytopenia, anaemia, elevated liver enzymes and decreases in albumin. 

Yondelis carries a warning alerting health care providers of the risk of severe and fatal neutropenic sepsis, rhabdomyolysis, hepatotoxicity, extravasation, tissue necrosis and cardiomyopathy. Patients with known hypersensitivity to trabectedin, should not be treated with Yondelis.

Health care providers are also encouraged to advise women of potential risks to a developing foetus when taking Yondelis. Women who are taking Yondelis should not breastfeed.

Yondelis is marketed by Janssen Products of Raritan, New Jersey.