During the FDA's priority review period, patients with soft tissue sarcoma who are unlikely to benefit from available therapies and who cannot participate in a clinical trial can apply to receive Yondelis through an FDA expanded access program (NCT00210665 Study).
The investigational antitumor agent Yondelis was originally derived from the sea squirt, Ecteinascidia turbinate, and is now manufactured synthetically.>The approval is based on the phase 3 ET743-SAR-3007 trial, which compared Yondelis with dacarbazine in more than 500 patients with liposarcoma and leiomyosarcoma who previously received anthracycline and ifosfamide or an anthracycline followed by one additional line of chemotherapy.
The primary outcome measure of the trial is overall survival (OS). Secondary outcome measures include progression-free survival (PFS), objective response rate, time to progression, duration of response, and safety. At this point, no data for the phase 3 study are available, with the drug’s manufacturer noting in a statement that it intends to present the results at a future date.“Today, we are one step closer to our goal of making another treatment option available for people living with advanced soft tissue sarcoma,” Denise Reinke, president and CEO of Sarcoma Alliance for Research through Collaboration, said in a statement when Janssen filed its New Drug Application. “People living with this type of cancer are in urgent need of new options to help treat their disease, and we welcome new medicines that may help make a difference in their lives.”
Results were previously published for a phase 2 study of Yondelis combined with dexamethasone in pretreated patients with recurrent advanced soft tissue sarcoma. Among 35 evaluable patients, 18 patients had stable disease and 1 patient had a partial response. Median OS and PFS were 10.2 months and 2.1 months, respectively. Twenty-one patients had moderate neutropenia. In most cases, grade 3/4 toxicities were successfully managed through dose reductions. Drug-related toxicities were linked to four deaths among patients receiving Yondelis.