Rare Disease Report

Two Potential Hepatocellular Carcinoma Treatments Get Orphan Drug Designations

AUGUST 16, 2017
Mathew Shanley
Singapore-based clinical-stage biotechnology company Lion TCR was granted 2 orphan drug designations today by the U.S. Food and Drug Administration (FDA) for T-cell therapies.
HBV-specific TCR redirected T-cell therapies against HCC with transient mRNA and the longer lasting DNA transduction technologies are both in development for the treatment of hepatocellular carcinoma (HCC).
HCC, often the result of a chronic hepatitis B infection, has a poor general prognosis with 5-year survival rates ranging from 3% for advanced cancer to 31% for localized forms. It is a primary malignancy of the liver, and occurs mostly in patients with chronic liver disease and cirrhosis. Presentation of it typically transpires in later stages with right-upper-quadrant pain, weight loss, and signs of decompensated liver disease.
"Profiting from US FDA's well established regulatory framework and the ODD grant of our HBV specific TCR-T cell products, we aim to develop the clinical program internationally under FDA IND covering multiple sites in US, Europe and Asia. It will significantly accelerate our product development and speed up the commercialization of our products to both US and Asian markets,” said company founder and CEO, Dr. Li Lietao in a press release.
All T-cell therapies are transmitted to patients via intravenous infusion. Because the most active ingredient is the patient’s own white blood cells, there is generally little or no toxicity.
While HCC is the world’s third-leading cause of death due to cancer, and second for China, the orphan drug designation was granted with the understanding that there are fewer than 200,000 people affected by it in the U.S. The designation provides incentives and that may include tax credits trials and user waiver fees, and also entitles Lion TCR to 7 years of market exclusivity in the U.S., should the drug be approved.
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