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news.Tvardi Therapeutics, a clinical-stage biopharmaceutical company, has announced receipt of orphan drug designation (ODD) from the US Food and Drug Administration (FDA) for its lead product, TTI-101, to treat hepatocellular carcinoma (HCC).
Image: Although HCC is the most common type of liver cancer, options to treat the disease are restricted. Credit: Michal Jarmoluk from Pixabay
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Although HCC is the most common type of liver cancer, options to treat the disease are restricted.
The total prognosis for survival is poor, with a five-year survival rate being 18%.
Orally delivered, TTI-101 is a small molecule, direct inhibitor of STAT3, which is a primary regulatory protein that plays a crucial role in the pathogenesis of the disease by commencing tumorigenesis as well as promoting an immunosuppressive tumour microenvironment.
Tvardi CEO Imran Alibhai said: “We are pleased to receive Orphan Drug Designation for TTI-101 in HCC from the FDA.
“This designation builds on the compelling safety and efficacy we have seen in last-line HCC patients in our ongoing Phase 1 trial.
“We look forward to the impending initiation of our Phase 2 trial in HCC to test TTI-101 as monotherapy as well as in combination.”
The receipt of ODD will enable Tvardi to get partial tax credits for qualified HCC clinical studies, receive benefit from exemptions from user fees for new drug applications and qualify the programme on the lead product for seven years of market exclusivity after approval from FDA.
Currently, TTI-101 is under investigation in a Phase 1 single agent multicentre clinical study in patients detected with advanced solid tumours.
