Oncternal Therapeutics Receives Rare Pediatric Disease Designation from U.S. Food and Drug Administration for TK216 for Treatment of Ewing Sarcoma
The FDAs rare pediatric disease designation of TK216 for treatment of Ewing sarcoma, for which Oncternal had previously received FDAs Orphan Drug and Fast Track designations, underscores the agencys recognition that Ewing sarcoma is a devastating cancer, with a high unmet medical need, said James Breitmeyer, M.D., Ph.D., President and CEO, Oncternal.
- The FDAs rare pediatric disease designation of TK216 for treatment of Ewing sarcoma, for which Oncternal had previously received FDAs Orphan Drug and Fast Track designations, underscores the agencys recognition that Ewing sarcoma is a devastating cancer, with a high unmet medical need, said James Breitmeyer, M.D., Ph.D., President and CEO, Oncternal.
- The prognosis for patients with recurrent Ewing sarcoma is particularly poor, and five-year survival after recurrence is approximately 10 to 15%.
- Tumorigenic fusion proteins involving the EWS protein and an ETS protein can be found in most cases of Ewing sarcoma.
- The U.S. Food and Drug Administration (FDA) has granted Orphan designation, Fast Track designation, and Rare Pediatric Disease designation to TK216 for the treatment of Ewing sarcoma.