Yondelis(trabectedin)

Yondelis (trabectedin) is indicated for the treatment of patients with advanced soft tissue sarcoma who have failed prior anthracycline and ifosfamide treatment, or who are unsuitable for these agents. It received marketing authorization on 17 September 2007 and has orphan designation EU/3/03/171 and EU/3/01/039. Trabectedin is approved in 75 countries for soft tissue sarcoma. The estimated new cases of soft tissue sarcoma in 2015 were 11,930.

In a randomized, open-label pivotal study, Yondelis demonstrated a median progression-free survival of 4.2 months compared to 1.5 months in the comparator arm, with a hazard ratio for progression-free survival of 0.55 (p-value <0.001). The Objective Response Rate was 7%. Median overall survival was 13.7 months compared to 13.1 months, with a hazard ratio of 0.93 (p-value 0.49). The median Duration of Response was 6.9 months. For the Leiomyosarcoma subgroup, the HR for PFS was 0.55.

Safety findings include myocardial dysfunction (11% vs 5% in placebo), pneumothorax (3% vs 0% in placebo), and venous thromboembolism (5% vs 2% in placebo). A total of 378 patients received at least one dose of trabectedin, with 197 patients exposed for at least 6 months and 57 patients for at least 1 year.