Sarcomas are a group of aggressive cancers of connective tissue in the body for which there are currently limited treatment options. In 2010, the American Cancer Society estimated that approximately 10,600 new cases of soft-tissue sarcomas would be diagnosed in the United States, with more than 3,900 Americans expected to die of the disease. In addition, approximately 2,600 new cases of bone sarcomas were expected to be diagnosed and nearly 1,500 deaths were estimated. Sadly, there has been no new approved therapy in the U.S. for patients with soft-tissue or bone sarcomas in more than 20 years.
Ridaforolimus has been designated both as a fast-track and orphan drug product by the U.S. Food and Drug Administration (FDA) and as an orphan drug by the European Medicines Agency (EMEA) for the treatment of soft-tissue and bone sarcomas.
Ridaforolimus: a highly targeted new approach.
Phase 3 SUCCEED clinical trial in sarcoma
January 2011, ARIAD announced that the Phase 3 SUCCEED trial of ridaforolimus met its primary endpoint—improved progression-free survival (PFS) compared with a placebo—in patients with metastatic soft-tissue and bone sarcomas who have had a favorable response to chemotherapy.
Both in independent committee and investigative site analyses, ridaforolimus showed statistically significant reductions in risk of progression and median PFS. Full press release here.
Merck is pursuing this indication as the initial registration path for ridaforolimus, and has submitted a new drug application (NDA) for ridaforolimus to the U.S. Food and Drug Administration.