FDA grants fast track designation to TT-00420 for cholangiocarcinoma
The FDA granted fast track designation to TT-00420 for treatment of patients with cholangiocarcinoma who have no standard therapy options.
TT-00420 (TransThera Sciences) is a spectrum selective kinase inhibitor.
Preclinical experiments showed the agent has high potency to a variety of FGFR2 mutations. It has demonstrated activity against cholangiocarcinoma, triple-negative breast cancer and other malignant tumors.
The FDA based the fast track designation on results of a phase 1 study that included nine patients with cholangiocarcinoma treated with TT-00420 at various dose levels.
Seven patients had at least one post-treatment efficacy assessment. Five achieved stable disease and two achieved partial response.
Five of the nine patients with cholangiocarcinoma harbored FGFR2 fusion or rearrangement and developed acquired resistance to prior FGFR inhibitor therapy. One of those patients achieved partial response, remaining progression free for about 10 months. The other four achieved stable disease.
“Receiving fast track designation is an important milestone for the development of TT-00420,” Frank Wu, PhD, CEO of TransThera, said in a company-issued press release. “We ... will continue to actively work with FDA, expediting the clinical development of TT-00420 in cholangiocarcinoma.”
The FDA previously granted orphan drug designation to TT-00420 for cholangiocarcinoma treatment.
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