FDA approves Yondelis for LPS, LMS

FDA recently approved trabectedin (Yondelis, Janssen Biotech) for the treatment of patients with unresectable or metastatic liposarcoma (LPS) or leiomyosarcoma (LMS) who received a prior anthracycline-containing regimen.

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While approved for both LPS and LMS, Yondelis is the first treatment to be specifically approved for LPS in the United States.

“A high unmet medical need remains for LPS and LMS patients. LPS and LMS are complex, rare and aggressive diseases and patients need new, FDA-approved treatment options,” Bernadette L. King, director of product communications – oncology in Janssen’s Global Pharmaceuticals Communication & Public Affairs office, told FormularyWatch.

The price for Yondelis, given by infusion every 3 weeks, is $8,100 per infusion. “Based on clinical data, patients received approximately 4 to 5 infusions, and as such, average drug costs per course of therapy are no more than $40,500,” King said.

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Since Yondelis was first approved in Europe in 2007, approximately 50,000 patients in close to 80 countries have benefited from this therapy, according to Janssen.

The new drug approval was based on recently published clinical efficacy and safety data from a phase 3, randomized, open-label, controlled study, which evaluated Yondelis versus the chemotherapy agent dacarbazine in patients with unresectable or metastatic LPS or LMS previously treated with an anthracycline and at least one additional chemotherapy regimen.

 The study included more than 500 patients and demonstrated a signficiant 45% improvement in progression free survival (PFS) for patients treated with Yondelis. The median PFS among the Yondelis treatment group was 4.2 months, while the median PFS in the dacarbazine treatment group was 1.5 months.

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The overall survival rate was a median of 13.7 months for Yondelis patients versus 13.1 months for those taking dacarbazine, which was not significant.

“In the clinical trial, Yondelis significantly increased progression free survival compared to dacarbazine; this is an important endpoint for these patients, in whom rapid worsening of the disease can lead to worse symptoms and life-threatening situations,” said George D. Demetri, MD, director of the Ludwig Center at Harvard and Director of the Center for Sarcoma and Bone Oncology at the Dana-Farber Cancer Institute and principal investigator of the phase 3 registration trial.

The most serious risks associated with Yondelis are neutropenic sepsis (severe infections due to decreased white blood cells), rhabdomyolysis (severe muscle problems), cardiomyopathy (heart muscle problems, including heart failure), hepatotoxicity (liver problems, including liver failure), anaphylaxis, and extravasation (leakage of Yondelis out of the vein during infusion) leading to tissue necrosis (tissue cell damage or death) and embryofetal toxicity.