The U.S. Food and Drug Administration granted Orphan Drug Designation to TK216 for the treatment of Ewing sarcoma, according to an announcement by Oncternal Therapeutics.1

TK216 is a first-in-class small molecule that inhibits ets-family transcription factor oncoproteins, the key disease drivers in Ewing sarcoma tumors. The treatment halts cancer cell growth and tumor formation.

“Ewing sarcoma is a rare pediatric cancer, and most patients who present with metastatic disease or fail first-line therapy face a poor prognosis. These young patients are in urgent need of better treatment options,” said James Breitmeyer, MD, PhD, Oncternal’s President and CEO, in a press release.

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Oncternal is recruiting patients to establish the initial safety and efficacy of intravenous TK216 for patients aged 12 years or older with relapsed or refractory Ewing sarcoma, in an open-label, phase 1, dose-escalation study. Final data collection for assessing the primary outcome measures is expected to be completed in late 2017.

“We are pleased to have support from the FDA through the Orphan Drug Designation program as we initiate our first clinical trial of TK216, treating relapsed or refractory Ewing sarcoma patients 12 years and older,” Dr Breitmeyer said.

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Orphan Drug Designation is designed to provide tax credit and fee waivers to the developing company if the drug or biologic agent meets predefined criteria and treats, diagnoses, or prevents a disease or disorder that affects fewer than 200,000 individuals in the United States.           


  1. Oncternal Therapeutics’ TK216 receives Orphan Drug Designation for the treatment of Ewing sarcoma, a rare pediatric cancer. PR Newswire. Updated July 13, 2016. Accessed July 20, 2016.