The US Food and Drug Administration (FDA) granted TT-00420, a potential new treatment for cholangiocarcinoma, Fast Track Designation, as announced in a press release by TransThera Sciences (Nanjing) Inc., the developers of the treatment.

Currently, the only potentially curative treatment option for cholangiocarcinoma is surgery. However, some patients are not eligible for surgery. 

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TT-00420 is a spectrum-selective kinase inhibitor that targets tumor cells and improves the tumor microenvironment. It received Fast Track Designation for the treatment of patients who have no standard treatment options.

Preclinical studies have shown TT-00420 can inhibit a number of malignant tumors, including cholangiocarcinoma and triple-negative breast cancer.

The Fast Track Designation is based on the results of a phase 1 clinical trial where 9 patients with cholangiocarcinoma were treated with TT-00420. Of these, 2 had a partial response to the treatment and 5 achieved stable disease. The other 2 patients were not evaluable with the treatment efficacy assessment. 

“Receiving Fast Track Designation is an important milestone for the development of TT-00420,” Frank Wu, DO, the chief executive officer of TransThera, said in the press release. “We have been and will continue to actively work with FDA, expediting the clinical development of TT-00420 in [the cholangiocarcinoma] field.”

The treatment had already received Orphan Drug Designation from the FDA for the treatment of cholangiocarcinoma in November 2020.

The Fast Track Designation will facilitate the development and expedite the review of TT-00420, meaning it could get to patients who need it faster than it normally would.

A new phase 2 clinical trial will soon start recruiting participants aged 18 years and over with cholangiocarcinoma to evaluate the safety and efficacy of TT-00420.


TransThera receives Fast Track Designation from FDA for its core product TT-00420 to treat cholangiocarcinoma. News release. TransThera Sciences (Nanjing) Inc.; November 3, 2021. 

Study to evaluate the efficacy and safety of TT-00420 in cholangiocarcinoma. June 9, 2021. Updated October 26, 2021. Accessed November 9, 2021.