This article is more than 5 years old. Information may no longer be current.

FDA grants TK216 orphan drug designation for Ewing sarcoma

Add topic to email alerts

Receive an email when new articles are posted on

Please provide your email address to receive an email when new articles are posted on .

Subscribe

Added to email alerts

You've successfully added to your alerts. You will receive an email when new content is published.

Click Here to Manage Email Alerts

You've successfully added to your alerts. You will receive an email when new content is published.

Click Here to Manage Email Alerts

Back to Healio

We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com.

Back to Healio

The FDA today granted orphan drug designation to TK216 for the treatment of Ewing sarcoma, a rare pediatric cancer comprised of highly malignant, undifferentiated tumors of bone.

Oncternal Therapeutics, the manufacturer of TK216, has initiated the first-in-human phase 1 trial of TK216, a first-in-class small molecule inhibiting ets-family transcription factor oncoproteins. The trial will include patients with relapsed or refractory Ewing sarcoma.

“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,” James Breitmeyer, MD, PhD, president and CEO of Oncternal, said in a company release. “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."

The FDA also granted TK216 fast track designation for this indication.