The FDA has recently approved a drug, TK216, for the management of Ewing Sarcoma (ES) which is a rare pediatric cancer. Most patients who are suffering from ES usually present after the disease has metastasized or after the failure of first-line of therapy and hence show a poor prognosis. The TK216 can inhibit the ets -family transcription factor oncoproteins that are the primary drivers of the disease.

The FDA has granted TK216 an Orphan Drug Designation and phase 1, open-label, dose-escalation studies are now underway to study the efficacy and safety of the drug in patients who are 10 years or older and have relapsed ES. The results of the trial are estimated in 2019. Source: NIH Clinical Trials, Cancer Therapy Advisor